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1.
Nat Med ; 27(3): 401-410, 2021 03.
Artículo en Inglés | MEDLINE | ID: mdl-33723456

RESUMEN

The twenty-first century has already recorded more than ten major epidemic or pandemic virus emergence events, including the ongoing and devastating coronavirus disease 2019 (COVID-19) pandemic. As viral disease emergence is expected to accelerate, these data dictate a need for proactive approaches to develop broadly active family-specific and cross-family therapeutics for use in future disease outbreaks. Emphasis should focus not only on the development of broad-spectrum small-molecule and antibody direct-acting antivirals, but also on host-factor therapeutics, including repurposing previously approved or in-pipeline drugs. Another new class of therapeutics with great antiviral therapeutic potential is RNA-based therapeutics. Rather than only focusing on known risks, dedicated efforts must be made toward pre-emptive research focused on outbreak-prone virus families, ultimately offering a strategy to shorten the gap between outbreak and response. Emphasis should also focus on orally available drugs for outpatient use, if possible, and on identifying combination therapies that combat viral and immune-mediated pathologies, extend the effectiveness of therapeutic windows and reduce drug resistance. While such an undertaking will require new vision, dedicated funding and private, federal and academic partnerships, this approach offers hope that global populations need never experience future pandemics such as COVID-19.


Asunto(s)
Enfermedades Transmisibles Emergentes/terapia , Terapias en Investigación , Virosis/terapia , Antivirales/uso terapéutico , /epidemiología , Desarrollo de Medicamentos/métodos , Desarrollo de Medicamentos/tendencias , Reposicionamiento de Medicamentos , Historia del Siglo XXI , Humanos , Invenciones/tendencias , Pandemias , Terapias en Investigación/métodos , Terapias en Investigación/tendencias
2.
Arch Med Res ; 52(1): 25-37, 2021 01.
Artículo en Inglés | MEDLINE | ID: mdl-33334622

RESUMEN

Nonalcoholic fatty liver disease (NAFLD) is the most common chronic liver disease, with an estimated rising prevalence, in concert with the epidemics of obesity and type 2 diabetes. The pathogenesis of NAFLD is not fully elucidated. Besides weight gain and insulin resistance, many other factors seem to contribute, including adipokines, gut microbiota and genetic predisposition. The disease starts as hepatic steatosis, which may proceed to nonalcoholic steatohepatitis (NASH); if fibrosis is added, the risk of cirrhosis and/or hepatocellular carcinoma is augmented. Liver biopsy is considered the gold standard for the diagnosis and staging of NAFLD; the early use of reliable and easily applied diagnostic tools, such as noninvasive biomarkers, is needed to identify patients at different-preferably early-stages of disease however. Whilst lifestyle modification is the first step to manage NAFLD, there is poor compliance, leading to the need of drug therapy. Accordingly, a variety of medications is under investigation. Given the multifaceted pathophysiology of NAFLD, probably, a combination of approaches in an individualized basis may be a more appropriate management. This review summarizes evidence on the epidemiology, pathogenesis, diagnosis and treatment of NAFLD.


Asunto(s)
Enfermedad del Hígado Graso no Alcohólico , Biomarcadores/análisis , Biomarcadores/sangre , Carcinoma Hepatocelular/complicaciones , Carcinoma Hepatocelular/diagnóstico , Carcinoma Hepatocelular/epidemiología , Carcinoma Hepatocelular/terapia , Diabetes Mellitus Tipo 2/complicaciones , Diabetes Mellitus Tipo 2/diagnóstico , Diabetes Mellitus Tipo 2/epidemiología , Diabetes Mellitus Tipo 2/terapia , Humanos , Estilo de Vida , Cirrosis Hepática/diagnóstico , Cirrosis Hepática/epidemiología , Cirrosis Hepática/etiología , Cirrosis Hepática/terapia , Neoplasias Hepáticas/complicaciones , Neoplasias Hepáticas/diagnóstico , Neoplasias Hepáticas/epidemiología , Neoplasias Hepáticas/terapia , Enfermedad del Hígado Graso no Alcohólico/diagnóstico , Enfermedad del Hígado Graso no Alcohólico/epidemiología , Enfermedad del Hígado Graso no Alcohólico/etiología , Enfermedad del Hígado Graso no Alcohólico/terapia , Obesidad/complicaciones , Obesidad/diagnóstico , Obesidad/epidemiología , Obesidad/terapia , Prevalencia , Factores de Riesgo , Terapias en Investigación/métodos , Terapias en Investigación/tendencias
3.
Swiss Med Wkly ; 150: w20446, 2020 12 14.
Artículo en Inglés | MEDLINE | ID: mdl-33382449

RESUMEN

AIMS OF THE STUDY: Hydroxychloroquine and lopinavir/ritonavir have been used as experimental therapies to treat COVID-19 during the first wave of the pandemic. Randomised controlled trials have recently shown that there are no meaningful benefits of these two therapies in hospitalised patients. Uncertainty remains regarding the potential harmful impact of these therapies as very early treatments and their burden to the health care system. The present study investigated the length of hospital stay (LOS), mortality, and costs of hydroxychloroquine, lopinavir/ritonavir or their combination in comparison with standard of care among patients hospitalised for coronavirus disease 2019 (COVID-19). METHODS: This retrospective observational cohort study took place in the Geneva University Hospitals, Geneva, Switzerland (n = 840) between 26 February and 31 May 2020. Demographics, treatment regimens, comorbidities, the modified National Early Warning Score (mNEWS) on admission, and contraindications to COVID-19 treatment options were assessed. Outcomes included LOS, in-hospital mortality, and drug and LOS costs. RESULTS: After successful propensity score matching, patients treated with (1) hydroxychloroquine, (2) lopinavir/ritonavir or (3) their combination had on average 3.75 additional hospitalisation days (95% confidence interval [CI] 1.37–6.12, p = 0.002), 1.23 additional hospitalisation days (95% CI −1.24 – 3.51, p = 0.319), and 4.19 additional hospitalisation days (95% CI 1.52–5.31, p <0.001), respectively, compared with patients treated with the standard of care. Neither experimental therapy was significantly associated with mortality. These additional hospital days amounted to 1010.77 additional days for hydroxychloroquine and hydroxychloroquine combined with lopinavir/ritonavir, resulting in an additional cost of US$ 2,492,214 (95%CI US$ 916,839–3,450,619). CONCLUSIONS: Prescribing experimental therapies for COVID-19 was not associated with a reduced LOS and might have increased the pressure put on healthcare systems.


Asunto(s)
Antivirales/uso terapéutico , /epidemiología , Hidroxicloroquina/uso terapéutico , Lopinavir/uso terapéutico , Ritonavir/uso terapéutico , Adolescente , Adulto , Factores de Edad , Anciano , Anciano de 80 o más Años , Antivirales/administración & dosificación , Antivirales/efectos adversos , Niño , Preescolar , Comorbilidad , Combinación de Medicamentos , Quimioterapia Combinada , Gastos en Salud , Mortalidad Hospitalaria/tendencias , Humanos , Hidroxicloroquina/administración & dosificación , Hidroxicloroquina/efectos adversos , Lactante , Tiempo de Internación/estadística & datos numéricos , Lopinavir/administración & dosificación , Lopinavir/efectos adversos , Persona de Mediana Edad , Pandemias , Estudios Retrospectivos , Ritonavir/administración & dosificación , Ritonavir/efectos adversos , Índice de Severidad de la Enfermedad , Factores Sexuales , Factores Socioeconómicos , Terapias en Investigación/métodos , Adulto Joven
4.
Trials ; 21(1): 853, 2020 Oct 15.
Artículo en Inglés | MEDLINE | ID: mdl-33059771

RESUMEN

OBJECTIVES: To evaluate the efficacy of two doses of the adsorbed vaccine COVID-19 (inactivated) produced by Sinovac in symptomatic individuals, with virological confirmation of COVID-19, two weeks after the completion of the two-dose vaccination regimen, aged 18 years or older who work as health professionals providing care to patients with possible or confirmed COVID-19. To describe the occurrence of adverse reactions associated with the administration of each of two doses of the adsorbed vaccine COVID-19 (inactivated) produced by Sinovac up to one week after vaccination in Adults (18-59 years of age) and Elderly (60 years of age or more). TRIAL DESIGN: This is a Phase III, randomized, multicenter, endpoint driven, double-blind, placebo-controlled clinical trial to assess the efficacy and safety of the adsorbed vaccine COVID-19 (inactivated) produced by Sinovac. The adsorbed vaccine COVID-19 (inactivated) produced by Sinovac (product under investigation) will be compared to placebo. Voluntary participants will be randomized to receive two intramuscular doses of the investigational product or the placebo, in a 1: 1 ratio, stratified by age group (18 to 59 years and 60 years or more) and will be monitored for one year by active surveillance of COVID-19. Two databases will be established according to the age groups: one for adults (18-59 years) and one for the elderly (60 years of age or older). The threshold to consider the vaccine efficacious will be to reach a protection level of at least 50%, as proposed by the World Health Organization and the FDA. Success in this criterion will be defined by sequential monitoring with adjustment of the lower limit of the 95% confidence interval above 30% for the primary efficacy endpoint. PARTICIPANTS: Healthy participants and / or participants with clinically controlled disease, of both genders, 18 years of age or older, working as health professionals performing care in units specialized in direct contact with people with possible or confirmed cases of COVID-19. Participation of pregnant women and those who are breastfeeding, as well as those intending to become pregnant within three months after vaccination will not be allowed. Participants will only be included after signing the voluntary Informed Consent Form and ensuring they undergo screening evaluation and conform to all the inclusion and exclusion criteria. All the clinical sites are located in Brazil. INTERVENTION AND COMPARATOR: Experimental intervention: The vaccine was manufactured by Sinovac Life Sciences (Beijing, China) and contains 3 µg/0.5 mL (equivalent to 600 SU per dose) of inactivated SARS-CoV-2 virus, and aluminium hydroxide as adjuvant. Control comparator: The placebo contains aluminium hydroxide in a 0.5 mL solution The schedule of both, experimental intervention and placebo is two 0.5 mL doses IM (deltoid) with a two week interval. MAIN OUTCOMES: The primary efficacy endpoint is the incidence of symptomatic cases of virologically confirmed COVID-19 two weeks after the second vaccination. The virological diagnosis will be confirmed by detection of SARS-CoV-2 nucleic acid in a clinical sample. The primary safety endpoint is the frequency of solicited and unsolicited local and systemic adverse reactions during the period of one week after vaccination according to age group in adult (18-59 years old) and elder (60 years of age or older) subjects. Adverse reactions are defined as adverse events that have a reasonable causal relationship to vaccination. RANDOMISATION: There will be two randomization lists, one for each age group, based on the investigational products to be administered, i.e., vaccine or placebo at a 1: 1 ratio. Each randomization list will be made to include up to 11,800 (18-59 year-old) adults, and 1,260 elderly (60 y-o and older) participants, the maximum number of participants needed per age group. An electronic central randomization system will be used to designate the investigational product that each participant must receive. BLINDING (MASKING): This trial is designed as a double-blind study to avoid introducing bias in the evaluation of efficacy, safety and immunogenicity. The clinical care team, the professionals responsible for the vaccination and the participants will not know which investigational product will be administered. Only pharmacists or nurses in the study who are responsible for the randomization, separation and blinding of the investigational product will have access to unblinded information. The sponsor's operational team will also remain blind. NUMBERS TO BE RANDOMISED (SAMPLE SIZE): The total number of participants needed to evaluate efficacy, 13,060 participants, satisfies the needed sample size calculated to evaluate safety. Therefore, the total number obtained for efficacy will be the number retained for the study. Up to 13,060 participants are expected to enter the study, with up to 11,800 participants aged 18 to 59 years and 1,260 elderly participants aged 60 and over. Half of the participants of each group will receive the experimental vaccine and half of them will receive the placebo. The recruitment of participants may be modified as recommended by the Data Safety Monitoring Committee at time of the interim unblinded analysis or blind assessment of the COVID-19 attack rate during the study. TRIAL STATUS: Protocol version 2.0 - 24-Aug-2020. Recruitment started on July 21st, 2020. The recruitment is expected to conclude in October 2020. TRIAL REGISTRATION: ClinicalTrials.gov Identifier: NCT0445659 . Registry on 2 July 2020 FULL PROTOCOL: The full protocol is attached as an additional file, accessible from the Trials website (Additional file 1). In the interest in expediting dissemination of this material, the familiar formatting has been eliminated; this Letter serves as a summary of the key elements of the full protocol.


Asunto(s)
Betacoronavirus/genética , Infecciones por Coronavirus/prevención & control , Pandemias/prevención & control , Neumonía Viral/prevención & control , Vacunación/métodos , Vacunas/uso terapéutico , Adolescente , Adulto , Anciano , Betacoronavirus/inmunología , Brasil/epidemiología , Estudios de Casos y Controles , Infecciones por Coronavirus/epidemiología , Infecciones por Coronavirus/inmunología , Infecciones por Coronavirus/virología , Manejo de Datos , Método Doble Ciego , Femenino , Personal de Salud/estadística & datos numéricos , Humanos , Incidencia , Consentimiento Informado/ética , Inyecciones Intramusculares , Masculino , Persona de Mediana Edad , Placebos/administración & dosificación , Neumonía Viral/epidemiología , Neumonía Viral/inmunología , Neumonía Viral/virología , Seguridad , Terapias en Investigación/métodos , Resultado del Tratamiento , Vacunas/administración & dosificación , Vacunas/efectos adversos , Adulto Joven
5.
J Alzheimers Dis ; 77(4): 1805-1813, 2020.
Artículo en Inglés | MEDLINE | ID: mdl-32986671

RESUMEN

BACKGROUND: The COVID-19 pandemic has brought great disruption to health systems worldwide. This affected ongoing clinical research, particularly among those most vulnerable to the pandemic, like dementia patients. Fundació ACE is a research center and memory clinic based in Barcelona, Spain, one of the hardest-hit countries. OBJECTIVE: To describe the ad-hoc strategic plan developed to cope with this crisis and to share its outcomes. METHODS: We describe participants' clinical and demographic features. Additionally, we explain our strategic plan aimed at minimizing the impact on clinical trial research activities, which included SARS-CoV-2 RT-PCR and IgG serological tests to all participants and personnel. The outcomes of the plan are described in terms of observed safety events and drop-outs during the study period. RESULTS: A total of 130 patients were participating in 16 active clinical trials in Fundació ACE when the lockdown was established. During the confinement, we performed 1018 calls to the participants, which led to identify adverse events in 26 and COVID-19 symptoms in 6. A total of 83 patients (64%) could restart on-site visits as early as May 11, 2020. All SARS-CoV-2 RT-PCR diagnostic tests performed before on-site visits were negative and only three IgG serological tests were positive. Throughout the study period, we only observed one drop-out, due to an adverse event unrelated to COVID-19. DISCUSSION: The plan implemented by Fundació ACE was able to preserve safety and integrity of ongoing clinical trials. We must use the lessons learned from the pandemic and design crisis-proof protocols for clinical trials.


Asunto(s)
Enfermedad de Alzheimer/terapia , Ensayos Clínicos como Asunto , Infecciones por Coronavirus , Pandemias , Atención al Paciente , Neumonía Viral , Anciano , Instituciones de Atención Ambulatoria , Betacoronavirus , Técnicas de Laboratorio Clínico , Ensayos Clínicos como Asunto/métodos , Ensayos Clínicos como Asunto/organización & administración , Infecciones por Coronavirus/diagnóstico , Infecciones por Coronavirus/epidemiología , Infecciones por Coronavirus/prevención & control , Femenino , Humanos , Masculino , Pandemias/prevención & control , Atención al Paciente/métodos , Atención al Paciente/tendencias , Neumonía Viral/epidemiología , Neumonía Viral/prevención & control , España/epidemiología , Telemedicina/métodos , Terapias en Investigación/métodos
9.
Med Sci (Paris) ; 36(8-9): 725-734, 2020.
Artículo en Francés | MEDLINE | ID: mdl-32821049

RESUMEN

The prognosis for phenylketonuria (PKU) has been improved by neonatal screening and dietary management via a low-phenylalanine diet. This treatment must be followed throughout life, which induces severe compliance problems. Drug treatment with sapropterin (or BH4) has come to help a reduced percentage of patients who respond to this drug. A subcutaneous enzyme therapy is available in the USA and has obtained European marketing authorization, but generates significant side effects, which limits its effectiveness. New therapeutic options for PKU are currently being developed, in particular gene therapy. The purpose of this article is to take stock of the pathophysiology and the various new therapeutic modalities currently in development.


Asunto(s)
Dieta , Terapia Genética , Fenilcetonurias/terapia , Biopterina/análogos & derivados , Biopterina/uso terapéutico , Dieta/efectos adversos , Dieta/métodos , Terapia Genética/métodos , Terapia Genética/tendencias , Humanos , Recién Nacido , Tamizaje Neonatal , Fenilcetonurias/diagnóstico , Fenilcetonurias/dietoterapia , Pronóstico , Terapias en Investigación/métodos , Terapias en Investigación/tendencias
10.
Presse Med ; 49(3): 104035, 2020 Oct.
Artículo en Inglés | MEDLINE | ID: mdl-32645417

RESUMEN

Immunoglobulin A vasculitis (IgAV, formerly Henoch-Schönlein purpura) is a systemic inflammatory disease affecting small vessels. While it is common and usually benign in childhood, in adults it is rarer has a more severe course. Its main manifestations are cutaneous purpura, arthralgias or arthritis, acute enteritis and glomerulonephritis. Renal involvement is associated with a poor prognosis in adults. The treatment of adult-onset IgAV is still a matter of debate: although in patients with a non-severe phenotype remission can occur spontaneously, more severe cases may need immunosuppressive therapy. There are some areas of uncertainty with respect to the efficacy of immunosuppressive regimens: almost all data come from studies performed in children or from patients with IgA nephropathy and/or IgA-crescentic glomerulonephritis. The only randomised study performed in adults with IgAV and renal involvement showed that immunosuppressive therapy with cyclophosphamide did not improve renal outcome nor did it affect patient survival. The possible efficacy of other drugs is reported only in small case series. Recent evidences show that rituximab could be an effective therapeutic option for adult-onset IgAV, but this also needs to be confirmed in controlled trials. In this review, we focus on therapeutic options for adult-onset IgAV treatment, and discuss the main results of the studies performed so far.


Asunto(s)
Púrpura de Schoenlein-Henoch/terapia , Terapias en Investigación/tendencias , Adulto , Edad de Inicio , Cardiología/métodos , Cardiología/tendencias , Niño , Glomerulonefritis por IGA/tratamiento farmacológico , Glomerulonefritis por IGA/patología , Hematología/métodos , Hematología/tendencias , Humanos , Inmunoglobulina A/efectos adversos , Inmunoglobulina A/inmunología , Inmunosupresores/uso terapéutico , Púrpura de Schoenlein-Henoch/epidemiología , Púrpura de Schoenlein-Henoch/patología , Rituximab/uso terapéutico , Terapias en Investigación/métodos
11.
Can J Physiol Pharmacol ; 98(8): 483-489, 2020 Aug.
Artículo en Inglés | MEDLINE | ID: mdl-32640179

RESUMEN

In response to the outbreak of severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), researchers are expeditiously searching for antiviral treatments able to alleviate the symptoms of infection, which can be life-threatening. Here, we provide a general overview of what is currently known about the structure and characteristic features of SARS-CoV-2, some of which could potentially be exploited for the purposes of antiviral therapy and vaccine development. This minireview also covers selected and noteworthy antiviral agents/supportive therapy out of hundreds of drugs that are being repurposed or tested as potential treatments for COVID-19, the disease caused by SARS-CoV-2.


Asunto(s)
Antivirales/farmacología , Betacoronavirus , Infecciones por Coronavirus , Pandemias , Neumonía Viral , Terapias en Investigación/métodos , Betacoronavirus/aislamiento & purificación , Betacoronavirus/patogenicidad , Betacoronavirus/fisiología , Infecciones por Coronavirus/tratamiento farmacológico , Infecciones por Coronavirus/epidemiología , Infecciones por Coronavirus/terapia , Humanos , Neumonía Viral/epidemiología , Neumonía Viral/terapia , Resultado del Tratamiento
12.
J Mycol Med ; 30(3): 101007, 2020 Sep.
Artículo en Inglés | MEDLINE | ID: mdl-32718789

RESUMEN

Mucormycosis are life-threatening fungal infections especially affecting immunocompromised or diabetic patients. Despite treatment, mortality remains high (from 32 to 70% according to organ involvement). This review provides an update on mucormycosis management. The latest recommendations strongly recommend as first-line therapy the use of liposomal amphotericin B (≥5mg/kg) combined with surgery whenever possible. Isavuconazole and intravenous or delayed-release tablet forms of posaconazole have remained second-line. Many molecules are currently in development to fight against invasive fungal diseases but few have demonstrated efficacy against Mucorales. Despite in vitro efficacy, combinations of treatment have failed to demonstrate superiority versus monotherapy. Adjuvant therapies are particularly complex to evaluate without prospective randomized controlled studies, which are complex to perform due to low incidence rate and high mortality of mucormycosis. Perspectives are nonetheless encouraging. New approaches assessing relationships between host, fungi, and antifungal drugs, and new routes of administration such as aerosols could improve mucormycosis treatment.


Asunto(s)
Infectología/normas , Infectología/tendencias , Mucormicosis/terapia , Guías de Práctica Clínica como Asunto , Anfotericina B/uso terapéutico , Antifúngicos/uso terapéutico , Terapia Combinada/métodos , Terapia Combinada/normas , Terapia Combinada/tendencias , Complicaciones de la Diabetes/microbiología , Humanos , Huésped Inmunocomprometido , Infectología/métodos , Procedimientos Quirúrgicos Operativos/métodos , Procedimientos Quirúrgicos Operativos/normas , Procedimientos Quirúrgicos Operativos/tendencias , Terapias en Investigación/métodos , Terapias en Investigación/tendencias
13.
Int J Cardiol ; 318: 160-164, 2020 11 01.
Artículo en Inglés | MEDLINE | ID: mdl-32610153

RESUMEN

The novel coronavirus disease, affecting ~9 million people in the past five months and causing >460,000 deaths worldwide, is completely new to mankind. More than 2,000 research projects registered at ClinTrials.gov are aiming at finding effective treatments for rapid transfer to clinical practice. Unfortunately, just few studies have a sufficiently valid design to provide reliable information for clinical practice.


Asunto(s)
Antivirales/farmacología , Ensayos Clínicos como Asunto , Infecciones por Coronavirus , Reposicionamiento de Medicamentos/métodos , Pandemias , Neumonía Viral , Terapias en Investigación/métodos , Betacoronavirus , Ensayos Clínicos como Asunto/métodos , Ensayos Clínicos como Asunto/organización & administración , Ensayos Clínicos como Asunto/normas , Infecciones por Coronavirus/tratamiento farmacológico , Infecciones por Coronavirus/epidemiología , Humanos , Italia , Neumonía Viral/tratamiento farmacológico , Neumonía Viral/epidemiología , Investigación , Proyectos de Investigación
14.
J Couns Psychol ; 67(4): 409-419, 2020 Jul.
Artículo en Inglés | MEDLINE | ID: mdl-32614223

RESUMEN

In recent years, innovative approaches have been implemented in counseling and psychotherapy research, creating new and exciting interdisciplinary subfields. The findings that emerged from the implementation of these approaches demonstrate their potential to deepen our understanding of therapeutic change. This article serves as an introduction to the "Innovative Approaches to Exploring Processes of Change in Counseling Psychology" special issue. The special issue includes articles representing several of the most promising approaches. Each article seeks to serve as a sourcebook for implementing a given approach in counseling research, in such areas as the assessment of coregulation processes, language processing, physiology, motion synchrony, event-related potentials, hormonal measures, and sociometric signals captured by a badge. The studies included in this special issue represent some of the most promising pathways for future studies and provide valuable resources for researchers, as well as clinicians interested in implementing such approaches and/or being educated consumers of empirical findings based on such approaches. This introduction synthesizes the articles in the special issue and proposes a list of guidelines for conducting and consuming research that implements new approaches for studying the process of therapeutic change. We believe that we are not far from the day when these approaches will be instrumental in everyday counseling practice, where they can assist therapists and patients in their collaborative efforts to reduce suffering and increase thriving. (PsycInfo Database Record (c) 2020 APA, all rights reserved).


Asunto(s)
Investigación Biomédica/tendencias , Consejo/tendencias , Psicoterapia/tendencias , Investigación Biomédica/métodos , Comprensión , Consejo/métodos , Predicción , Humanos , Psicoterapia/métodos , Terapias en Investigación/métodos , Terapias en Investigación/tendencias
16.
Med Sci (Paris) ; 36(6-7): 600-606, 2020.
Artículo en Francés | MEDLINE | ID: mdl-32614311

RESUMEN

In inherited retinal diseases such retinitis pigmentosa, characterized by progressive loss of light sensitive neurons (photoreceptors), cell therapy is now considered as an attractive strategy. Photoreceptor cell replacement would be valuable for restoring function to retinas in a way that is independent from the cause of the disease. With advances in stem cell biology, considerable strides have been made towards the generation of retinal cells, in particular with the development of 3D culture systems allowing the generation of retinal organoids from pluripotent stem cells. In this review, we present a state-of-the art of preclinical strategies conducted in animal models for photoreceptor replacement from stem cell-derived photoreceptors and we discuss the important obstacles to overcome in the future.


Asunto(s)
Células Fotorreceptoras/trasplante , Retinitis Pigmentosa/terapia , Terapias en Investigación/métodos , Terapias en Investigación/tendencias , Animales , Humanos , Células Madre Pluripotentes Inducidas/citología , Células Madre Pluripotentes Inducidas/trasplante , Organoides/citología , Organoides/fisiología , Células Fotorreceptoras/citología , Células Fotorreceptoras/fisiología , Células Madre Pluripotentes/citología , Células Madre Pluripotentes/trasplante , Retina/citología , Retina/embriología , Retina/trasplante , Degeneración Retiniana/terapia , Retinitis Pigmentosa/patología , Índice de Severidad de la Enfermedad , Técnicas de Cultivo de Tejidos , Recolección de Tejidos y Órganos/métodos , Recolección de Tejidos y Órganos/normas , Recolección de Tejidos y Órganos/tendencias
17.
Med Sci (Paris) ; 36(6-7): 616-625, 2020.
Artículo en Francés | MEDLINE | ID: mdl-32614313

RESUMEN

Iron has a fundamental role for cell physiology and especially in retina as a cofactor of many pathways of the visual transduction. A tightly regulated homeostasis avoids the accumulation of prooxidant and proinflammatory free iron. A dysfunction of iron retinal homeostasis is associated with many genetic or age-related degenerative diseases such as age-related macular degeneration (AMD). Here, we describe various mechanisms reported during AMD, enhanced by iron accumulation and its homeostasis dysregulation. We have investigated a local treatment with transferrin, the natural iron carrier, to control these pathological pathways and iron dysfunction, without side effects. Iron has a central role in pathogenesis of AMD and is a target for futures therapies.


Asunto(s)
Hierro/fisiología , Degeneración Macular/etiología , Homeostasis/genética , Humanos , Hierro/metabolismo , Degeneración Macular/genética , Degeneración Macular/metabolismo , Degeneración Macular/terapia , Redes y Vías Metabólicas/genética , Retina/metabolismo , Retina/patología , Terapias en Investigación/métodos , Terapias en Investigación/tendencias , Transferrina/genética , Transferrina/fisiología
18.
Med Sci (Paris) ; 36(6-7): 626-632, 2020.
Artículo en Francés | MEDLINE | ID: mdl-32614314

RESUMEN

Generation of retinal organoids from pluripotent stem cells represents an important advance in the study of retinal development and offer new perspectives for the study of retinal diseases missing suitable animal models. Understanding the key stages of retinal development in vertebrates enabled to design protocols to generate self-organized three-dimensional structures derived from pluripotent stem cells and containing all retinal cell types. In addition to their application in basic research, such as the characterization of molecular and cellular mechanisms in retinal pathophysiology, these miniature organs also open up encouraging prospects in the field of cell therapy or the screening of therapeutic molecules, although some obstacles remain to be overcome.


Asunto(s)
Organoides/citología , Retina/citología , Enfermedades de la Retina/etiología , Enfermedades de la Retina/patología , Enfermedades de la Retina/terapia , Animales , Células Cultivadas , Humanos , Modelos Biológicos , Organoides/fisiología , Retina/patología , Retina/fisiología , Terapias en Investigación/métodos , Terapias en Investigación/tendencias , Técnicas de Cultivo de Tejidos/métodos , Técnicas de Cultivo de Tejidos/tendencias
20.
Eur J Endocrinol ; 183(2): G67-G77, 2020 Aug.
Artículo en Inglés | MEDLINE | ID: mdl-32508313

RESUMEN

The COVID-19 pandemic is a major international emergency leading to unprecedented medical, economic and societal challenges. Countries around the globe are facing challenges with diabetes care and are similarly adapting care delivery, with local cultural nuances. People with diabetes suffer disproportionately from acute COVID-19 with higher rates of serious complications and death. In-patient services need specialist support to appropriately manage glycaemia in people with known and undiagnosed diabetes presenting with COVID-19. Due to the restrictions imposed by the pandemic, people with diabetes may suffer longer-term harm caused by inadequate clinical support and less frequent monitoring of their condition and diabetes-related complications. Outpatient management need to be reorganised to maintain remote advice and support services, focusing on proactive care for the highest risk, and using telehealth and digital services for consultations, self-management and remote monitoring, where appropriate. Stratification of patients for face-to-face or remote follow-up should be based on a balanced risk assessment. Public health and national organisations have generally responded rapidly with guidance on care management, but the pandemic has created a tension around prioritisation of communicable vs non-communicable disease. Resulting challenges in clinical decision-making are compounded by a reduced clinical workforce. For many years, increasing diabetes mellitus incidence has been mirrored by rising preventable morbidity and mortality due to complications, yet innovation in service delivery has been slow. While the current focus is on limiting the terrible harm caused by the pandemic, it is possible that a positive lasting legacy of COVID-19 might include accelerated innovation in chronic disease management.


Asunto(s)
Betacoronavirus , Infecciones por Coronavirus/epidemiología , Infecciones por Coronavirus/terapia , Diabetes Mellitus/epidemiología , Diabetes Mellitus/terapia , Neumonía Viral/epidemiología , Neumonía Viral/terapia , Terapias en Investigación/tendencias , Infecciones por Coronavirus/diagnóstico , Diabetes Mellitus/diagnóstico , Endocrinología/métodos , Endocrinología/tendencias , Humanos , Pandemias , Neumonía Viral/diagnóstico , Telemedicina/métodos , Telemedicina/tendencias , Terapias en Investigación/métodos , Reino Unido/epidemiología
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