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1.
Can J Psychiatry ; 69(3): 217-227, 2024 03.
Artigo em Inglês | MEDLINE | ID: mdl-37644885

RESUMO

OBJECTIVE: This study aims to understand whether higher use of a patient portal can have an impact on mental health functioning and recovery. METHOD: A mixed methods approach was used for this study. In 2019-2021, patients with mental health diagnoses at outpatient clinics in an academic centre were invited to complete World Health Organization Disability Assessment Scale 12 (WHODAS-12) and Mental Health Recovery Measure surveys at baseline, 3 months, and 6 months after signing up for the portal. At the 3-month time point, patients were invited to a semistructured interview with a member of the team to contextualize the findings obtained from the surveys. Analytics data was also collected from the platform to understand usage patterns on the portal. RESULTS: Overall, 113 participants were included in the analysis. There was no significant change in mental health functioning and recovery scores over the 6-month period. However, suboptimal usage was observed as 46% of participants did not complete any tasks within the portal. Thirty-five participants had low use of the portal (1-9 interactions) and 18 participants had high usage (10+ interactions). There were also no differences in mental health functioning and recovery scores between low and high users of the portal. Qualitative interviews highlighted many opportunities where the portal can support overall functioning and mental health recovery. CONCLUSIONS: Collectively, this study suggests that higher use of a portal had no impact, either positive or negative, on mental health outcomes. While it may offer convenience and improved patient satisfaction, adequate support is needed to fully enable these opportunities for patient care. As the type of interaction with the portal was not specifically addressed, future work should focus on looking at ways to support patient engagement and portal usage throughout their care journey.


Assuntos
Saúde Mental , Portais do Paciente , Humanos , Inquéritos e Questionários , Satisfação do Paciente
2.
J Med Internet Res ; 25: e42507, 2023 02 10.
Artigo em Inglês | MEDLINE | ID: mdl-36763409

RESUMO

BACKGROUND: The increasing integration of digital health tools into care may result in a greater flow of personal health information (PHI) between patients and providers. Although privacy legislation governs how entities may collect, use, or share PHI, such legislation has not kept pace with digital health innovations, resulting in a lack of guidance on implementing meaningful consent. Understanding patient perspectives when implementing meaningful consent is critical to ensure that it meets their needs. Consent for research in the context of digital health is limited. OBJECTIVE: This state-of-the-art review aimed to understand the current state of research as it relates to patient perspectives on digital health consent. Its objectives were to explore what is known about the patient perspective and experience with digital health consent and provide recommendations on designing and implementing digital health consent based on the findings. METHODS: A structured literature search was developed and deployed in 4 electronic databases-MEDLINE, IEEE Xplore, Scopus, and Web of Science-for articles published after January 2010. The initial literature search was conducted in March 2021 and updated in March 2022. Articles were eligible for inclusion if they discussed electronic consent or consent, focused on the patient perspective or preference, and were related to digital health or digital PHI. Data were extracted using an extraction template and analyzed using qualitative content analysis. RESULTS: In total, 75 articles were included for analysis. Most studies were published within the last 5 years (58/75, 77%) and conducted in a clinical care context (33/75, 44%) and in the United States (48/75, 64%). Most studies aimed to understand participants' willingness to share PHI (25/75, 33%) and participants' perceived usability and comprehension of an electronic consent notice (25/75, 33%). More than half (40/75, 53%) of the studies did not describe the type of consent model used. The broad open consent model was the most explored (11/75, 15%). Of the 75 studies, 68 (91%) found that participants were willing to provide consent; however, their consent behaviors and preferences were context-dependent. Common patient consent requirements included clear and digestible information detailing who can access PHI, for what purpose their PHI will be used, and how privacy will be ensured. CONCLUSIONS: There is growing interest in understanding the patient perspective on digital health consent in the context of providing clinical care. There is evidence suggesting that many patients are willing to consent for various purposes, especially when there is greater transparency on how the PHI is used and oversight mechanisms are in place. Providing this transparency is critical for fostering trust in digital health tools and the innovative uses of data to optimize health and system outcomes.


Assuntos
Registros de Saúde Pessoal , Privacidade , Humanos , Estados Unidos , Pacientes , Confiança , Consentimento Livre e Esclarecido
3.
BMC Nurs ; 21(1): 213, 2022 Aug 04.
Artigo em Inglês | MEDLINE | ID: mdl-35927701

RESUMO

BACKGROUND: Although EHR systems have become a critical part of clinical care, nurses are experiencing a growing burden due to documentation requirements, taking time away from other important clinical activities. There is a need to address the inefficiencies and challenges that nurses face when documenting in and using EHRs. The objective of this study is to engage nurses in generating ideas on how organizations can support and optimize nurses' experiences with their EHR systems, thereby improving efficiency and reducing EHR-related burden. This work will ensure the identified solutions are grounded in nurses' perspectives and experiences and will address their specific EHR-related needs. METHODS: This mixed methods study will consist of three phases. Phase 1 will evaluate the accuracy of the EHR system's analytics platform in capturing how nurses utilize the system in real-time for tasks such as documentation, chart review, and medication reconciliation. Phase 2 consists of a retrospective analysis of the nursing-specific analytics platform and focus groups with nurses to understand and contextualize their usage patterns. These focus groups will also be used to identify areas for improvement in the utilization of the EHR. Phase 3 will include focus groups with nurses to generate and adapt potential interventions to address the areas for improvement and assess the perceived relevance, feasibility, and impact of the potential interventions. DISCUSSION: This work will generate insights on addressing nurses' EHR-related burden and burnout. By understanding and contextualizing inefficiencies and current practices, opportunities to improve EHR systems for nursing professional practice will be identified. The study findings will inform the co-design and implementation of interventions that will support adoption and impact. Future work will include the evaluation of the developed interventions, and research on scaling and disseminating the interventions for use in different organizations, EHR systems, and jurisdictions in Canada.

4.
J Med Internet Res ; 23(10): e30491, 2021 10 19.
Artigo em Inglês | MEDLINE | ID: mdl-34665141

RESUMO

BACKGROUND: The COVID-19 pandemic has increased the demand for youth mental health services in Canada as disruptions to clinical care continue to persist due to the risk of transmission and exposure to the virus. Digital mental health interventions, including web-based resources and mobile apps, have provided opportunities to support youth mental health remotely across Canada. There is a need to better understand how these digital interventions are being selected, recommended, and used in various regions across Canada. OBJECTIVE: A national jurisdictional scan was completed to (1) determine what web-based programs, apps, and websites are promoted and licensed in Canada for youth mental health; (2) identify criteria and decision-making processes that Canadian jurisdictions use to select web-based programs, apps, and websites for youth mental health; and (3) identify upcoming trends, innovations, and digital mental health possibilities that are emerging in the youth sector. METHODS: The aims of the jurisdictional scan were addressed through a review of related academic and grey literature; stakeholder interviews, including individuals involved in various areas of the youth mental health sector; and a social media review of pertinent Twitter content. RESULTS: A total of 66 web-based resources and apps were identified for use by youth in Canada. 16 stakeholder interviews were completed and included discussions with researchers, clinicians, youth organizations, and others involved in digital interventions for youth mental health. These discussions identified a limited use of frameworks used to guide decision-making processes when selecting digital interventions. Many clinicians agreed on a similar set of eligibility requirements for youth mental health apps and digital resources, such as the evidence base and cultural relevance of the intervention. Stakeholders also identified upcoming trends and innovations in the youth digital mental health space, including artificial intelligence, digital phenotyping, and personalized therapy. Over 4 weeks, 2184 tweets were reviewed to identify and compare global and national trends and innovations involving digital mental health and youth. Key trends included the promotion of regional chat services as well as the effects of the COVID-19 pandemic on youth mental health and access to care. CONCLUSIONS: As organizations begin to plan for the delivery of mental health care following the pandemic, there are concerns about the sustainability of these digital mental health interventions as well as a need for services to be more informed by the experiences and preferences of youth.


Assuntos
COVID-19 , Saúde Mental , Adolescente , Inteligência Artificial , Canadá , Tomada de Decisão Clínica , Humanos , Pandemias , SARS-CoV-2
5.
Bioconjug Chem ; 31(3): 673-684, 2020 03 18.
Artigo em Inglês | MEDLINE | ID: mdl-31986014

RESUMO

Targeting the inability of cancerous cells to adapt to metabolic stress is a promising alternative to conventional cancer chemotherapy. FTY720 (Gilenya), an FDA-approved drug for the treatment of multiple sclerosis, has recently been shown to inhibit cancer progression through the down-regulation of essential nutrient transport proteins, selectively starving cancer cells to death. However, the clinical use of FTY720 for cancer therapy is prohibited because of its capability of inducing immunosuppression (lymphopenia) and bradycardia when phosphorylated upon administration. A prodrug to specifically prevent phosphorylation during circulation, hence avoiding bradycardia and lymphopenia, was synthesized by capping its hydroxyl groups with polyethylene glycol (PEG) via an acid-cleavable ketal linkage. Improved aqueous solubility was also accomplished by PEGylation. The prodrug reduces to fully potent FTY720 upon cellular uptake and induces metabolic stress in cancer cells. Enhanced release of FTY720 at a mildly acidic endosomal pH and the ability to substantially down-regulate cell-surface nutrient transporter proteins in leukemia cells only by an acid-cleaved drug were confirmed. Importantly, the prodrug demonstrated nearly identical efficacy to FTY720 in an animal model of BCR-Abl-driven leukemia without inducing bradycardia or lymphopenia in vivo, highlighting its potential clinical value. The prodrug formulation of FTY720 demonstrates the utility of precisely engineering a drug to avoid undesirable effects by tackling specific molecular mechanisms as well as a financially favorable alternative to new drug development. A multitude of existing cancer therapeutics may be explored for prodrug formulation to avoid specific side effects and preserve or enhance therapeutic efficacy.


Assuntos
Materiais Biocompatíveis/química , Materiais Biocompatíveis/farmacologia , Cloridrato de Fingolimode/química , Cloridrato de Fingolimode/farmacologia , Leucemia/tratamento farmacológico , Polietilenoglicóis/química , Acetais/química , Antineoplásicos/química , Antineoplásicos/farmacologia , Linhagem Celular Tumoral , Humanos , Concentração de Íons de Hidrogênio , Leucemia/patologia , Fosforilação
6.
J Med Internet Res ; 22(7): e19274, 2020 07 15.
Artigo em Inglês | MEDLINE | ID: mdl-32673234

RESUMO

BACKGROUND: Physician burnout has a direct impact on the delivery of high-quality health care, with health information technology tools such as electronic health records (EHRs) adding to the burden of practice inefficiencies. OBJECTIVE: The aim of this study was to determine the extent of burnout among physicians and learners (residents and fellows); identify significant EHR-related contributors of physician burnout; and explore the differences between physicians and learners with regard to EHR-related factors such as time spent in EHR, documentation styles, proficiency, training, and perceived usefulness. In addition, the study aimed to address gaps in the EHR-related burnout research methodologies by determining physicians' patterns of EHR use through usage logs. METHODS: This study used a cross-sectional survey methodology and a review of administrative data for back-end log measures of survey respondents' EHR use, which was conducted at a large Canadian academic mental health hospital. Chi-square and Fisher exact tests were used to examine the association of EHR-related factors with general physician burnout. The survey was sent out to 474 individuals between May and June 2019, including physicians (n=407), residents (n=53), and fellows (n=14), and we measured physician burnout and perceptions of EHR stressors (along with demographic and practice characteristics). RESULTS: Our survey included 208 respondents, including physicians (n=176) and learners (n=32). The response rate was 43.2% for physicians (full-time: 156/208, 75.0%; part-time: 20/199, 10.1%), and 48% (32/67) for learners. A total of 25.6% (45/176) of practicing physicians and 19% (6/32) of learners reported having one or more symptoms of burnout, and 74.5% (155/208) of all respondents who reported burnout symptoms identified the EHR as a contributor. Lower satisfaction and higher frustration with the EHRs were significantly associated with perceptions of EHR contributing toward burnout. Physicians' and learners' experiences with the EHR, gathered through open-ended survey responses, identified challenges around the intuitiveness and usability of the technology as well as workflow issues. Metrics gathered from back-end usage logs demonstrated a 13.6-min overestimation in time spent on EHRs per patient and a 5.63-hour overestimation of after-hours EHR time, when compared with self-reported survey data. CONCLUSIONS: This study suggests that the use of EHRs is a perceived contributor to physician burnout. There should be a focus on combating physician burnout by reducing the unnecessary administrative burdens of EHRs through efficient implementation of systems and effective postimplementation strategies.


Assuntos
Esgotamento Profissional/psicologia , Registros Eletrônicos de Saúde/normas , Médicos/psicologia , Adulto , Estudos Transversais , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Inquéritos e Questionários
7.
J Med Internet Res ; 22(3): e16263, 2020 03 06.
Artigo em Inglês | MEDLINE | ID: mdl-32141833

RESUMO

BACKGROUND: Compassion is a vital component to the achievement of positive health outcomes, particularly in mental health care. The rise of digital technologies may influence the delivery of compassionate care, and thus this relationship between compassion and digital health care needs to be better understood. OBJECTIVE: This scoping review aimed to identify existing digital technologies being used by patients and health professionals in the delivery of mental health care, understand how digital technologies are being used in the delivery of compassionate mental health care, and determine the facilitators of and barriers to digital technology use among patients and health professionals in the delivery of compassionate mental health care. METHODS: We conducted this scoping review through a search of Cumulative Index to Nursing and Allied Health Literature, Medical Literature Analysis and Retrieval System Online (MEDLINE), MEDLINE In-Process and EPub Ahead of Print, PsycINFO, and Web of Science for articles published from 1990 to 2019. RESULTS: Of the 4472 articles screened, 37 articles were included for data extraction. Telemedicine was the most widely used technology by mental health professionals. Digital technologies were described as facilitating compassionate care and were classified using a conceptual model to identify each digital intersection with compassionate care. Facilitators of and barriers to providing compassionate care through digital technology were identified, including increased safety for providers, health care professional perceptions and abilities, and the use of picture-in-picture feedback to evaluate social cues. CONCLUSIONS: Implementing digital technology into mental health care can improve the current delivery of compassionate care and create novel ways to provide compassion. However, as this is a new area of study, mental health professionals and organizations alike should be mindful that compassionate human-centered care is maintained in the delivery of digital health care. Future research could develop tools to facilitate and evaluate the enactment of compassion within digital health care.


Assuntos
Empatia/fisiologia , Saúde Mental/normas , Telemedicina/normas , Humanos
8.
Healthc Q ; 23(3): 54-62, 2020 Oct.
Artigo em Inglês | MEDLINE | ID: mdl-33243367

RESUMO

The use of electronic health record (EHR) systems has led to numerous discussions about their possible contribution to clinician burnout. However, discussions regarding effective strategies to reduce burnout of this nature have been few. This article reviews initiatives and strategies aimed at combatting EHR-related burnout and provides recommendations for Canadian contexts. While approaches for measuring and reducing EHR-related physician burnout were identified, this paper highlights a paucity of evidence surrounding EHR-associated burnout among non-physician clinicians and the efficacy of interventions aimed at reducing burnout. Based on the findings, this article proposes recommendations for optimizing EHR usage to potentially reduce burnout.


Assuntos
Esgotamento Profissional/prevenção & controle , Registros Eletrônicos de Saúde , Médicos/psicologia , Esgotamento Profissional/epidemiologia , Canadá , Humanos
9.
Stud Health Technol Inform ; 312: 101-106, 2024 Feb 19.
Artigo em Inglês | MEDLINE | ID: mdl-38372319

RESUMO

This paper maps suicide help-seeking needs identified in the literature, on to the features and functionalities of suicide prevention mobile apps using the adapted ecological model, thereby revealing existing gaps between help-seeking needs and available apps. This paper builds upon previous work by our team, which includes 1) a rapid scoping review aimed at identifying barriers and facilitators of help-seeking related to suicide within psychiatric populations, and 2) a review of suicide prevention apps, including a content analysis of app features and functionalities.


Assuntos
Serviços de Saúde Mental , Aplicativos Móveis , Suicídio , Telemedicina , Humanos , Prevenção do Suicídio , Suicídio/psicologia
10.
Stud Health Technol Inform ; 312: 16-21, 2024 Feb 19.
Artigo em Inglês | MEDLINE | ID: mdl-38372305

RESUMO

In partnership with clinician advisors, a text-based program, BeWell, was co-created to support clinician well-being at a Canadian mental health hospital. This paper briefly describes the process of designing BeWell with clinician advisors and highlights key lessons learned in engaging clinicians as advisors in the design and development of a digital health intervention. The lessons learned can serve as best practices for health systems, organizations, and researchers to consider when engaging clinicians in the design, development, and implementation of digital health interventions.


Assuntos
Saúde Mental , Envio de Mensagens de Texto , Canadá , Saúde Digital , Organizações
11.
Nurs Leadersh (Tor Ont) ; 35(4): 42-54, 2023 Jan.
Artigo em Inglês | MEDLINE | ID: mdl-37216296

RESUMO

The purpose of this paper is to describe a nursing informatics engagement strategy at an academic teaching hospital in Canada aimed at sustaining and retaining the nursing workforce by (1) enhancing nursing engagement and leadership in informatics decision making; (2) improving nurses' experiences using the electronic health record (EHR) by creating a process of rapid handling of technology issues; (3) leveraging data about nurses' EHR system use to identify opportunities to further streamline documentation; and (4) enhancing and optimizing informatics education/training and communication strategies. The nursing informatics strategy aims to improve engagement among nursing staff, as well as decrease the burden of using the EHR as a way of addressing possible causes of burnout.


Assuntos
Informática em Enfermagem , Recursos Humanos de Enfermagem , Humanos , Informática em Enfermagem/educação , Hospitais , Comunicação , Recursos Humanos
12.
AMIA Annu Symp Proc ; 2022: 1012-1021, 2022.
Artigo em Inglês | MEDLINE | ID: mdl-37128401

RESUMO

Clinician informatics leadership has been identified as an essential component of addressing the 'implementation to benefits realization gap' that exists for many digital health technologies. Chief Medical Informatics Officers (CMIOs), and Chief Nursing Informatics Officers (CNIOs) are well-positioned to ensure the success of these initiatives. However, while the CMIO role is fairly well-established in Canada, there is limited uptake of CNIO roles in the country. The main objective of this work is to build on the current progress of the CMIO role and explore how the CNIO role can be best positioned for uptake and value across healthcare organizations in Canada. A qualitative study was conducted. Ten clinician leaders in CMIO, CNIO, and related roles in Canada were interviewed about the value of these roles and strategies for supporting the uptake of the role. This study provides the foundation for future initiatives for supporting and showcasing the value of the CNIO in a digitally enabled healthcare organization.


Assuntos
Informática Médica , Informática em Enfermagem , Humanos , Canadá , Liderança
13.
Nano Converg ; 8(1): 34, 2021 Nov 02.
Artigo em Inglês | MEDLINE | ID: mdl-34727233

RESUMO

Modern medicine has been waging a war on cancer for nearly a century with no tangible end in sight. Cancer treatments have significantly progressed, but the need to increase specificity and decrease systemic toxicities remains. Early diagnosis holds a key to improving prognostic outlook and patient quality of life, and diagnostic tools are on the cusp of a technological revolution. Nanotechnology has steadily expanded into the reaches of cancer chemotherapy, radiotherapy, diagnostics, and imaging, demonstrating the capacity to augment each and advance patient care. Nanomaterials provide an abundance of versatility, functionality, and applications to engineer specifically targeted cancer medicine, accurate early-detection devices, robust imaging modalities, and enhanced radiotherapy adjuvants. This review provides insights into the current clinical and pre-clinical nanotechnological applications for cancer drug therapy, diagnostics, imaging, and radiation therapy.

14.
Stud Health Technol Inform ; 284: 539-541, 2021 Dec 15.
Artigo em Inglês | MEDLINE | ID: mdl-34920590

RESUMO

The introduction of electronic health records (EHR) systems can have a profound impact on the daily clinical activities of nurses. Recent studies have suggested that the adoption of EHR systems may have contributed to burnout among nurses. This poster presentation will explore potential strategies to address the emerging issue of burnout associated with EHR systems in nurses.


Assuntos
Esgotamento Profissional , Registros Eletrônicos de Saúde , Esgotamento Profissional/prevenção & controle , Humanos
15.
Biomater Sci ; 8(23): 6718-6729, 2020 Dec 07.
Artigo em Inglês | MEDLINE | ID: mdl-33111729

RESUMO

Advances in bionanotechnology aim to develop smart nucleic acid delivery carriers with stimuli-responsive features to overcome challenges such as non-biodegradability, rapid clearance, immune response, and reaching intracellular targets. Peptide-based nanomaterials have become widely used in the field of gene and drug delivery due to their structural versatility and biomimetic properties. Particularly, polypeptide gene vectors that respond to biological stimuli, such as acidic intracellular environments, have promising applications in mediating efficient endosomal escape and drug release. Unfortunately, synthesis strategies for efficient polymerization of acid-labile peptides have been limited due to conditions that fail to preserve acid-degradable functional groups. Stable urethane derivatives of the acid-labile amino acid ketalized serine (kSer) were synthesized and polymerized to a high molecular weight under permissive conditions independent of elevated temperature, restrictive solvents, or an inert atmosphere. A new formulation strategy utilizing solvent-driven self-assembly of poly(kSer) peptides with small interfering RNA (siRNA) was developed, and the resulting poly(kSer)/siRNA complexes were further cross-linked for reinforced stability under physiological conditions. The complexes were highly monodisperse and precisely spherical in morphology, which has significant clinical implications in definitive biodistribution, cellular internalization, and intracellular trafficking patterns. Self-assembled, cross-linked poly(kSer)/siRNA complexes demonstrated efficient nucleic acid encapsulation, internalization, endosomal escape, and acid-triggered cargo release, tackling multiple hurdles in siRNA delivery. The acid-responsive polypeptides and solvent-driven self-assembly strategies demonstrated in this study could be applicable to developing other efficient and safe delivery systems for gene and drug delivery.


Assuntos
Interferência de RNA , RNA Interferente Pequeno , Serina , RNA Interferente Pequeno/metabolismo , Solventes , Distribuição Tecidual
16.
Int J Exerc Sci ; 11(4): 417-424, 2018.
Artigo em Inglês | MEDLINE | ID: mdl-29795733

RESUMO

This study compared body composition measurements in lean female athletes. The primary objective was to compare the accuracy of percent body fat (%BF) determined by bioelectrical impedance analysis (BIA), air-displacement plethysmography (ADP), and underwater weighing (UWW) in female Division I cheerleaders (n = 10 bases, 6 back-spots, and 12 flyers) from two universities. The secondary objective was to compare health risk predicted by %BF to body mass index (BMI) categorizations. UWW was considered the gold standard for assessing %BF. Pearson correlation coefficients were used to determine associations between methods. Repeated measures analysis of variance was used to identify differences in %BF by method. BIA, ADP, and UWW were highly correlated (r ≥ .828, p < .001 for all). However, %BF by BIA (20.0 ± 5.2%) and ADP (19.3 ± 6.0%) was higher than %BF by UWW (15.9 ± 4.1%, p < .001). Health risk was predicted less often when classified based on very lean (risky low) %BF levels by BIA and ADP than UWW (7.1%, 3.6%, and 21.4%, respectively). This finding suggests that, similar to female track-and-field athletes who also exhibit lean muscular physiques, %BF is overestimated by BIA and ADP in female cheerleaders and health risk associated with low %BF is underestimated when compared to UWW. In contrast, BMI was not associated with %BF by any method and no participants were classified as underweight by this measure. Thus, BMI should not be used to predict health risk in lean female athletes, such as collegiate cheerleaders.

17.
Prog Retin Eye Res ; 63: 107-131, 2018 03.
Artigo em Inglês | MEDLINE | ID: mdl-29097191

RESUMO

Retinitis Pigmentosa (RP) is a hereditary retinopathy that affects about 2.5 million people worldwide. It is characterized with progressive loss of rods and cones and causes severe visual dysfunction and eventual blindness in bilateral eyes. In addition to more than 3000 genetic mutations from about 70 genes, a wide genetic overlap with other types of retinal dystrophies has been reported with RP. This diversity of genetic pathophysiology makes treatment extremely challenging. Although therapeutic attempts have been made using various pharmacologic agents (neurotrophic factors, antioxidants, and anti-apoptotic agents), most are not targeted to the fundamental cause of RP, and their clinical efficacy has not been clearly proven. Current therapies for RP in ongoing or completed clinical trials include gene therapy, cell therapy, and retinal prostheses. Gene therapy, a strategy to correct the genetic defects using viral or non-viral vectors, has the potential to achieve definitive treatment by replacing or silencing a causative gene. Among many clinical trials of gene therapy for hereditary retinal diseases, a phase 3 clinical trial of voretigene neparvovec (AAV2-hRPE65v2, Luxturna) recently showed significant efficacy for RPE65-mediated inherited retinal dystrophy including Leber congenital amaurosis and RP. It is about to be approved as the first ocular gene therapy biologic product. Despite current limitations such as limited target genes and indicated patients, modest efficacy, and the invasive administration method, development in gene editing technology and novel gene delivery carriers make gene therapy a promising therapeutic modality for RP and other hereditary retinal dystrophies in the future.


Assuntos
Terapia Genética/métodos , Retinose Pigmentar/genética , Retinose Pigmentar/terapia , Pesquisa Biomédica , Estudos Clínicos como Assunto , Humanos , Biologia Molecular
19.
Adv Drug Deliv Rev ; 98: 3-18, 2016 Mar 01.
Artigo em Inglês | MEDLINE | ID: mdl-26546465

RESUMO

The dynamic and versatile nature of diseases such as cancer has been a pivotal challenge for developing efficient and safe therapies. Cancer treatments using a single therapeutic agent often result in limited clinical outcomes due to tumor heterogeneity and drug resistance. Combination therapies using multiple therapeutic modalities can synergistically elevate anti-cancer activity while lowering doses of each agent, hence, reducing side effects. Co-administration of multiple therapeutic agents requires a delivery platform that can normalize pharmacokinetics and pharmacodynamics of the agents, prolong circulation, selectively accumulate, specifically bind to the target, and enable controlled release in target site. Nanomaterials, such as polymeric nanoparticles, gold nanoparticles/cages/shells, and carbon nanomaterials, have the desired properties, and they can mediate therapeutic effects different from those generated by small molecule drugs (e.g., gene therapy, photothermal therapy, photodynamic therapy, and radiotherapy). This review aims to provide an overview of developing multi-modal therapies using nanomaterials ("combo" nanomedicine) along with the rationale, up-to-date progress, further considerations, and the crucial roles of interdisciplinary approaches.


Assuntos
Nanoestruturas/administração & dosagem , Neoplasias/terapia , Animais , Antineoplásicos/administração & dosagem , Antineoplásicos/uso terapêutico , Terapia Combinada , Sistemas de Liberação de Medicamentos , Humanos , Nanomedicina , Nanoestruturas/uso terapêutico
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