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1.
JNCI Cancer Spectr ; 6(1)2022 02.
Artigo em Inglês | MEDLINE | ID: mdl-35005429

RESUMO

Background: Dissemination and implementation (D&I) research is a key factor in the uptake and use of evidence-based cancer control interventions. National Cancer Institute (NCI)-designated cancer centers are ideal settings in which to further D&I knowledge. The purpose of this study was to summarize the characteristics of NCI-funded D&I science grants in the nation's cancer centers to understand the nature, extent, and opportunity for this key type of translational work. Methods: We used the National Institutes of Health Research Portfolio Online Reporting Tool to identify active NCI-funded grants in D&I science at NCI clinical cancer centers (n = 13) and comprehensive cancer centers (n = 51) as well as their academic affiliates. Active projects were eligible for inclusion if they 1) were awarded directly to an NCI cancer center or an academic or research affiliate, and 2) identified D&I content in the abstract. Portfolio data were collected in February 2021. Results: We identified 104 active NCI-funded D&I research or training grants across the 64 cancer centers; 57.8% of cancer centers had at least 1 NCI-funded D&I grant. Most awards (71.1%) were for research grants. Training grants constituted 29.1% of D&I-focused grants. Overall, 50.0% of grants (n = 52) concentrated on specific cancers. Almost two-thirds of grants (n = 68, 65.4%) had a stated health equity focus. Conclusions: More than one-half of NCI-designated cancer centers have active funding in D&I science, reflecting a substantial investment by NCI. There remains considerable room for further development, which would further support NCI's translational mission.


Assuntos
Institutos de Câncer/estatística & dados numéricos , Financiamento Governamental/estatística & dados numéricos , Ciência da Implementação , National Cancer Institute (U.S.) , Neoplasias , Pesquisa Translacional Biomédica/estatística & dados numéricos , Humanos , Neoplasias/prevenção & controle , Pesquisa Translacional Biomédica/economia , Estados Unidos
2.
PLoS One ; 16(12): e0261343, 2021.
Artigo em Inglês | MEDLINE | ID: mdl-34914775

RESUMO

Universities are important sources of knowledge and key members of the regional innovation system. The key problem in Chinese universities is the low efficiency of the scientific and technological (S&T) transformation, which limits the promotion of regional innovation and economic development. This article proposes the three-stage efficiency analytical framework, which regards it as a complex and interactive process. Avoiding the problem of considering the input and output of university S&T transformation as a "black box" and neglecting the links among different transformation stages. The super efficiency network SBM model is applied to the heterogeneous region of the Yangtze River Economic Belt. Empirical research proves that university S&T transformation has not been effectively improved and the scientific resources invested in universities have not been efficiently utilized in recent years. Generally, Despite the correlation between regional economy and transformation efficiency, the exclusive increase in resources is not enough. Regional openness and the quality of research talents are key factors for the application of technological innovation and technology marketization. Universities should not only pursue the number of research outputs but pay more attention to high-quality knowledge production to overcome difficulties in research achievements transformation.


Assuntos
Pesquisa Translacional Biomédica/economia , Pesquisa Translacional Biomédica/tendências , Universidades/tendências , China , Desenvolvimento Econômico/tendências , Eficiência , Humanos , Invenções/economia , Investimentos em Saúde , Conhecimento , Rios , Desenvolvimento Sustentável/tendências , Tecnologia/economia , Tecnologia/tendências , Universidades/economia
4.
PLoS One ; 16(8): e0255704, 2021.
Artigo em Inglês | MEDLINE | ID: mdl-34407104

RESUMO

BACKGROUND: Governments commonly fund research with specific applications in mind. Such mechanisms may facilitate 'research translation' but funders may employ strategies that can also undermine the integrity of both science and government. We estimated the prevalence and investigated correlates of funder efforts to suppress health behaviour intervention trial findings. METHODS: Our sampling frame was lead or corresponding authors of papers (published 2007-2017) included in a Cochrane review, reporting findings from trials of interventions to improve nutrition, physical activity, sexual health, smoking, and substance use. Suppression events were based on a previous survey of public health academics. Participants answered questions concerning seven suppression events in their efforts to report the trial, e.g., [I was…] "asked to suppress certain findings as they were viewed as being unfavourable." We also examined the association between information on study funder, geographical location, targeted health behaviour, country democracy rating and age of publication with reported suppression. FINDINGS: We received responses from 104 authors (50%) of 208 eligible trials, from North America (34%), Europe (33%), Oceania (17%), and other countries (16%). Eighteen percent reported at least one of the seven suppression events relating to the trial in question. The most commonly reported suppression event was funder(s) expressing reluctance to publish because they considered the results 'unfavourable' (9% reported). We found no strong associations with the subject of research, funding source, democracy, region, or year of publication. CONCLUSIONS: One in five researchers in this global sample reported being pressured to delay, alter, or not publish the findings of health behaviour intervention trials. Regulation of funder and university practices, establishing study registries, and compulsory disclosure of funding conditions in scientific journals, are needed to protect the integrity of public-good research.


Assuntos
Administração Financeira/tendências , Comportamentos Relacionados com a Saúde , Ensaios Clínicos Controlados Aleatórios como Assunto , Projetos de Pesquisa , Pesquisadores/economia , Pesquisa Translacional Biomédica/economia , Alcoolismo/prevenção & controle , Dieta Saudável , Europa (Continente) , Exercício Físico , Programas Governamentais/economia , Humanos , América do Norte , Medicina Preventiva/métodos , Saúde Sexual , Inquéritos e Questionários , Uso de Tabaco/prevenção & controle
5.
Acad Med ; 96(6): 836-841, 2021 06 01.
Artigo em Inglês | MEDLINE | ID: mdl-34031305

RESUMO

The National Institutes of Health (NIH) has prioritized efforts to increase diversity in the biomedical research workforce. NIH-funded institutional career development awards may serve as one mechanism to facilitate these efforts. In 2013, the Duke University KL2 program, an internal career development program funded by the National Center for Advancing Translational Sciences, set a goal to increase the number of investigators from underrepresented racial and ethnic groups (UREGs) to ≥ 50% of KL2 awardees. From 2013 to 2019, 133 KL2 applications were received, 38% from UREG investigators. Of the 21 scholars selected, 10 (47.6%) were UREG investigators; all were Black/African American. This represents a threefold increase in the proportion of UREG applications and a sixfold increase in the proportion of UREG KL2 scholars compared with Duke's previous KL2 cycles (2003-2012), during which only 13% of applicants and 8.3% of funded scholars were UREGs. Of the 12 KL2 scholars (7 UREG) who completed the program, 5 have received NIH funding as principal investigators of an external K award or R01, and 4 of them are UREG investigators; this constitutes a post-KL2 NIH funding success rate of 57% (4/7) for UREG scholars. Achieving this programmatic priority was facilitated by institutional support, clear communication of goals to increase the proportion of UREG KL2 awardees, and intentional strategies to identify and support applicants. Strategies included targeted outreach to UREG investigators, partnerships with other institutional entities, structured assistance for investigators with preparing their applications, and a KL2 program structure addressing common barriers to success for UREG investigators, such as lack of consistent mentorship, protected research time, and peer support. The authors' experience suggests that KL2 and other internal career development programs may represent a scalable, national strategy to increase diversity in the biomedical research workforce.


Assuntos
Distinções e Prêmios , Diversidade Cultural , Etnicidade , Pesquisadores/economia , Apoio à Pesquisa como Assunto , Pesquisa Translacional Biomédica/economia , Humanos , National Institutes of Health (U.S.) , North Carolina , Estados Unidos , Universidades
6.
Nutr. hosp ; 38(n.extr.1): 19-28, abr. 2021. tab
Artigo em Espanhol | IBECS | ID: ibc-201892

RESUMO

Investigar en nutrición clínica y dietética consiste en "realizar actividades intelectuales y experimentales de modo sistemático con el propósito de aumentar los conocimientos de esta materia". La investigación biomédica que se realiza en las unidades de nutrición clínica y dietética (UNCyD) debe ser clínico-traslacional, entendida como la forma más rápida de conseguir que los nuevos conocimientos científicos se transfieran no solamente a la investigación clínica sino también a la práctica asistencial. Cuando las UNCyD realizan investigación, independientemente del tipo y la extensión, alcanzan mayores cotas de calidad en la atención sanitaria que prestan a sus usuarios. La práctica clínica y la investigación científica son actividades que constituyen una acción continuada e indisoluble. Por ello, tanto los responsables de las gerencias sanitarias como los de las unidades de nutrición y todos sus miembros tienen la obligación de fomentar la investigación. En el artículo se realiza un breve repaso histórico del nacimiento de la nutrición clínica como disciplina científica, se nombra someramente la legislación que sustenta la investigación biomédica, se proponen las principales claves para potenciar la investigación en las UNCyD y, por último, se orienta sobre las principales vías de financiación y sobre cómo realizar la transferencia y difusión de los resultados


Research in clinical nutrition and dietetics consists of "carrying out intellectual and experimental activities in a systematic way with the purpose of increasing knowledge on this topic." The biomedical research carried out in clinical nutrition and dietetics units (UNCyDs) must be clinical-translational, which is understood as the fastest way to ensure that new scientific knowledge is transferred not only to clinical research but also to clinical practice. When UNCyDs conduct research, regardless of type and extent, they achieve higher quality standards in the health care they provide to their users. Clinical practice and scientific research are activities that constitute a continuous, indissoluble action. For this reason, both those responsible for health management and nutrition units and all their members have an obligation to promote research. In this article, a brief historical review of the birth of clinical nutrition as a scientific discipline is made, the legislation that supports biomedical research is briefly mentioned, the main keys to promote research in UNCyD are proposed, and finally orientation is offered on the main funding for biomedical research programs, and how to transfer and disseminate results


Assuntos
Humanos , Pesquisa Biomédica , Unidades Hospitalares , Pesquisa Translacional Biomédica/organização & administração , Ciências da Nutrição/economia , Pesquisa Translacional Biomédica/economia , Obtenção de Fundos/economia , Dietética
7.
Health Res Policy Syst ; 19(1): 5, 2021 Jan 18.
Artigo em Inglês | MEDLINE | ID: mdl-33461564

RESUMO

The COVID-19 pandemic has created urgent demand around the world for knowledge generation about a novel coronavirus, its transmission, and control, putting academic institutions at the frontline of politics. While many academic institutions are well poised to conduct research, there are well-documented barriers for these institutions, particularly in low- and middle-income countries (LMICs), to further conduct strategic synthesis and dissemination to promote knowledge utilization among policy-makers. These systemic barriers to knowledge translation (KT) pose significant challenges for academic institutions seeking to take advantage of unprecedented policy windows to inform evidence-based decision-making. Global health funding organizations should prioritize the support of academic institutions' activities along the KT pathway, including both knowledge generation and strategic dissemination, to improve knowledge uptake for decision-making to improve health. Institutional capacity-building initiatives for KT have the potential to profoundly impact responses to this and future pandemics.


Assuntos
COVID-19 , Fortalecimento Institucional , Países em Desenvolvimento , Saúde Global , Política de Saúde , Pandemias , Pesquisa Translacional Biomédica , Planejamento em Desastres , Prática Clínica Baseada em Evidências , Organização do Financiamento , Humanos , Conhecimento , Formulação de Políticas , Apoio à Pesquisa como Assunto , SARS-CoV-2 , Instituições Acadêmicas , Pesquisa Translacional Biomédica/economia
9.
Acad Med ; 96(4): 558-567, 2021 04 01.
Artigo em Inglês | MEDLINE | ID: mdl-33332904

RESUMO

PURPOSE: This qualitative study examined fiscal and administrative (i.e., pre- and post-award grants process) barriers and facilitators to community-engaged research among stakeholders across 4 Clinical and Translational Science Awards (CTSA) institutions. METHOD: A purposive sample of 24 key informants from 3 stakeholder groups-community partners, academic researchers, and research administrators-from the CTSA institutions at the University of North Carolina at Chapel Hill, Medical University of South Carolina, Vanderbilt University Medical Center, and Yale University participated. Semistructured interviews were conducted in March-July 2018, including questions about perceived challenges and best practices in fiscal and administrative processes in community-engaged research. Transcribed interviews were independently reviewed and analyzed using the Rapid Assessment Process to facilitate key theme and quote identification. RESULTS: Community partners were predominantly Black, academic researchers and research administrators were predominantly White, and women made up two-thirds of the overall sample. Five key themes were identified: level of partnership equity, partnership collaboration and communication, institutional policies and procedures, level of familiarity with varying fiscal and administrative processes, and financial management expectations. No stakeholders reported best practices for the institutional policies and procedures theme. Cross-cutting challenges included communication gaps between stakeholder groups; lack of or limits in supporting community partners' fiscal capacity; and lack of collective awareness of each stakeholder group's processes, procedures, and needs. Cross-cutting best practices centered on shared decision making and early and timely communication between all stakeholder groups in both pre- and post-award processes. CONCLUSIONS: Findings highlight the importance of equitable processes, triangulated communication, transparency, and recognizing and respecting different financial management cultures within community-engaged research. This work can be a springboard used by CTSA institutions to build on available resources that facilitate co-learning and discussions between community partners, academic researchers, and research administrators on fiscal readiness and administrative processes for improved community-engaged research partnerships.


Assuntos
Pesquisa Participativa Baseada na Comunidade/economia , Pesquisa Participativa Baseada na Comunidade/estatística & dados numéricos , Relações Comunidade-Instituição , Financiamento Governamental/economia , Pesquisa Translacional Biomédica/economia , Pesquisa Translacional Biomédica/estatística & dados numéricos , Universidades/estatística & dados numéricos , Adulto , Connecticut , Feminino , Financiamento Governamental/estatística & dados numéricos , Humanos , Masculino , Pessoa de Meia-Idade , North Carolina , Pesquisa Qualitativa , South Carolina , Tennessee , Estados Unidos
11.
Nature ; 586(7831): 683-692, 2020 10.
Artigo em Inglês | MEDLINE | ID: mdl-33116284

RESUMO

Starting with the launch of the Human Genome Project three decades ago, and continuing after its completion in 2003, genomics has progressively come to have a central and catalytic role in basic and translational research. In addition, studies increasingly demonstrate how genomic information can be effectively used in clinical care. In the future, the anticipated advances in technology development, biological insights, and clinical applications (among others) will lead to more widespread integration of genomics into almost all areas of biomedical research, the adoption of genomics into mainstream medical and public-health practices, and an increasing relevance of genomics for everyday life. On behalf of the research community, the National Human Genome Research Institute recently completed a multi-year process of strategic engagement to identify future research priorities and opportunities in human genomics, with an emphasis on health applications. Here we describe the highest-priority elements envisioned for the cutting-edge of human genomics going forward-that is, at 'The Forefront of Genomics'.


Assuntos
Pesquisa Biomédica/tendências , Genoma Humano/genética , Genômica/tendências , Saúde Pública/normas , Pesquisa Translacional Biomédica/tendências , Pesquisa Biomédica/economia , COVID-19/genética , Genômica/economia , Humanos , National Human Genome Research Institute (U.S.)/economia , Mudança Social , Pesquisa Translacional Biomédica/economia , Estados Unidos
14.
Clin Transl Sci ; 13(4): 818-825, 2020 07.
Artigo em Inglês | MEDLINE | ID: mdl-32216083

RESUMO

Since 2006, the US Congress has appropriated ~ 7 billion dollars in total toward the (CTSA) program of the National Institutes of Health (NIH), representing ~ 1.5% of the NIH total budget. There is no doubt this investment has led to substantial improvements in clinical and translational research, but the impact of these large NIH-sponsored awards to academic medical centers have largely been documented by anecdotal accomplishments. This paper provides a purely quantitative assessment of the impact of these awards on clinical trials registered on ClinicalTrials.gov. In particular, we find a dramatic increase in the number of registered clinical trials and clinical trial enrollment associated with the CTSA grant award. Additionally, the impact is shown to be magnified with the number of years of receiving CTSA funding.


Assuntos
Centros Médicos Acadêmicos/estatística & dados numéricos , Ensaios Clínicos como Assunto/estatística & dados numéricos , National Institutes of Health (U.S.)/economia , Apoio à Pesquisa como Assunto/estatística & dados numéricos , Pesquisa Translacional Biomédica/estatística & dados numéricos , Centros Médicos Acadêmicos/economia , Ensaios Clínicos como Assunto/economia , Apoio à Pesquisa como Assunto/economia , Pesquisa Translacional Biomédica/economia , Estados Unidos
15.
Sci Rep ; 10(1): 5039, 2020 03 19.
Artigo em Inglês | MEDLINE | ID: mdl-32193395

RESUMO

With >70,000 yearly publications using mouse data, mouse models represent the best engrained research system to address numerous biological questions across all fields of science. Concerns of poor study and microbiome reproducibility also abound in the literature. Despite the well-known, negative-effects of data clustering on interpretation and study power, it is unclear why scientists often house >4 mice/cage during experiments, instead of ≤2. We hypothesized that this high animal-cage-density  practice abounds in published literature because more mice/cage could be perceived as a strategy to reduce housing costs. Among other sources of 'artificial' confounding, including cyclical oscillations of the 'dirty-cage/excrement microbiome', we ranked by priority the heterogeneity of modern husbandry practices/perceptions across three professional organizations that we surveyed in the USA. Data integration (scoping-reviews, professional-surveys, expert-opinion, and 'implementability-score-statistics') identified Six-Actionable Recommendation Themes (SART) as a framework to re-launch emerging protocols and intuitive statistical strategies to use/increase study power. 'Cost-vs-science' discordance was a major aspect explaining heterogeneity, and scientists' reluctance to change. With a 'housing-density cost-calculator-simulator' and fully-annotated statistical examples/code, this themed-framework streamlines the rapid analysis of cage-clustered-data and promotes the use of 'study-power-statistics' to self-monitor the success/reproducibility of basic and translational research. Examples are provided to help scientists document analysis for study power-based sample size estimations using preclinical mouse data to support translational clinical trials, as requested in NIH/similar grants or publications.


Assuntos
Criação de Animais Domésticos , Animais de Laboratório , Abrigo para Animais , Camundongos , Microbiota , Reprodutibilidade dos Testes , Pesquisa Translacional Biomédica , Criação de Animais Domésticos/economia , Animais , Abrigo para Animais/economia , Tamanho da Amostra , Pesquisa Translacional Biomédica/economia
16.
Therapie ; 75(1): 7-12, 2020.
Artigo em Francês | MEDLINE | ID: mdl-31987590

RESUMO

The rising emergence of bacterial resistances has led to a crisis which threatens human, animal and environmental health. The impact of the emergency is enormous in terms of public health and economics. Although there is a global awareness of the warnings and programmes supporting innovative actions to combat fight against antibiotic resistance, it must be admitted that proposed new antibiotics fail to find the economic profitability necessary for them to reach the market and become available for patients and the community. Moreover, it is necessary to develop tools/indicators to define effective interventions against antibiotic resistance. The work of the think-tank reported in this article concentrated on two aspects of translational research: - prevention and the impact on health of the antibiotic resistance issue, and - the specific requirements of clinical research leading to innovation in the fight against antibiotic resistance. This article, which reflects the thoughts of a group of French experts, proposes directly operational solutions which could be rapidly implemented and radically transform the quality and quantity of our resources available for the combat.


Assuntos
Antibacterianos/farmacologia , Infecções Bacterianas/tratamento farmacológico , Pesquisa Translacional Biomédica/organização & administração , Animais , Infecções Bacterianas/microbiologia , Farmacorresistência Bacteriana , Humanos , Saúde Pública , Pesquisa Translacional Biomédica/economia
17.
BMJ ; 367: l5766, 2019 10 23.
Artigo em Inglês | MEDLINE | ID: mdl-31645328

RESUMO

OBJECTIVE: To determine the extent to which late stage development of new drugs relies on support from public funding. DESIGN: Cohort study. SETTING: All new drugs containing one or more new molecular entities approved by the US Food and Drug Administration (FDA) between January 2008 and December 2017 via the new drug application pathway. MAIN OUTCOME MEASURES: Patents or drug development histories documenting late stage research contributions by a public sector research institution or a spin-off company, as well as each drug's regulatory approval pathway and first-in-class designation. RESULTS: Over the 10 year study period, the FDA approved 248 drugs containing one or more new molecular entities. Of these drugs, 48 (19%) had origins in publicly supported research and development and 14 (6%) originated in companies spun off from a publicly supported research program. Drugs in these groups were more likely to receive expedited FDA approval (68% v 47%, P=0.005) or be designated first in class (45% v 26%, P=0.007), indicating therapeutic importance. CONCLUSIONS: A review of the patents associated with new drugs approved over the past decade indicates that publicly supported research had a major role in the late stage development of at least one in four new drugs, either through direct funding of late stage research or through spin-off companies created from public sector research institutions. These findings could have implications for policy makers in determining fair prices and revenue flows for these products.


Assuntos
Ensaios Clínicos como Assunto/economia , Aprovação de Drogas/economia , Setor Público/economia , Pesquisa Translacional Biomédica/economia , Ensaios Clínicos como Assunto/estatística & dados numéricos , Estudos de Coortes , Aprovação de Drogas/legislação & jurisprudência , Aprovação de Drogas/estatística & dados numéricos , Humanos , Patentes como Assunto/estatística & dados numéricos , Setor Público/estatística & dados numéricos , Pesquisa Translacional Biomédica/estatística & dados numéricos , Estados Unidos , United States Food and Drug Administration/legislação & jurisprudência , United States Food and Drug Administration/estatística & dados numéricos
19.
Curr Oncol ; 26(4): 272-284, 2019 08.
Artigo em Inglês | MEDLINE | ID: mdl-31548808

RESUMO

Background: A novel way to build capacity in knowledge translation (kt) is through kt-focused grant competitions. Since 2009, the Knowledge Translation Research Network (KT-Net) has had a cancer-related kt grants program. We undertook an evaluation of the program to determine if KT-Net was achieving its aims of building capacity in cancer kt, advancing the science of kt, building partnerships, and leveraging funding. Methods: An adapted framework guided the evaluation. Nine funded studies from 4 competitions were included. Semi-structured telephone interviews were held with researchers, stakeholders (including knowledge users), members of grant review panels, and experts in kt. Interview transcripts were audio-recorded, transcribed, and analyzed thematically. A review of proposal and report documents was also conducted. Results: Funded researchers indicated that the grant competition was an essential funding program for cancer kt research. Competitions were perceived to build capacity in cancer kt among early-career researchers and to encourage innovative cancer kt research for which alternative funding sources are limited. The grants program resulted in incremental gains in advancing the science of kt. Suggestions to improve the program included stronger partnerships between the funder and the provincial cancer-system organization to optimize the application of research that is relevant to the organization's strategic objectives. Conclusions: The grants program met many of its aims by providing cancer researchers with an opportunity to gain capacity in cancer kt and by making incremental advances in kt science. Suggestions to improve the program included closer partnerships between the funder and the cancer-system organization.


Assuntos
Neoplasias/terapia , Pesquisa Translacional Biomédica/economia , Estudos de Avaliação como Assunto , Humanos , Avaliação de Programas e Projetos de Saúde
20.
Circ Res ; 125(7): 720-724, 2019 09 13.
Artigo em Inglês | MEDLINE | ID: mdl-31513489

RESUMO

The deletion of Arginine 14 of the phosholamban gene (PLN p.R14del) is associated with the pathogenesis of an inherited form of cardiomyopathy with prominent arrhythmias. Patients carrying the PLN R14del mutation are at risk of developing dilated cardiomyopathy or arrhythmogenic right ventricular cardiomyopathy. Although the genetic etiology is well defined, the molecular mechanism underlying the pathogenesis of PLN R14del-cardiomyopathy is unknown. Our CURE PLaN network, funded by the Foundation Leducq, will bring together leading scientists, clinicians, and patients to elucidate the genotype-phenotype relationships in R14del cardiomyopathy with the ultimate goal of developing innovative disease-specific therapeutic modalities. With the generous support of the Leducq Foundation, our Transatlantic Network of Excellence consortium to cure Phospholamban (PLN)-induced cardiomyopathy (CURE-PLaN) unites 6 leading centers to address the current challenges associated with arrhythmogenic right ventricular cardiomyopathy/dilated cardiomyopathy (DCM) with an initial focus on PLN and development of effective treatments. The Network is led by Evangelia (Litsa) Kranias (University of Cincinnati) in the United States and Pieter A. Doevendans (Netherlands Heart Institute/UMC Utrecht NL) in Europe. The other US project leaders are Kevin Costa (Cardiovascular Research Center, Icahn School of Medicine at Mount Sinai, New York) and Mark Mercola and Ioannis Karakikes (Stanford University), who are focusing on induced pluripotent stem cell (iPSC)-based disease models, tissue engineering, gene therapy, and drug discovery. On the European side, the project leaders are Despina Sanoudou (Biomedical Research Foundation of the Academy of Athens) analyzing the PLN interactome and Stephan Lehnart (University of Gottingen) addressing the subcellular and disrupted protein interactions affected in PLN-mutant cardiomyocytes. Other key members within the Netherlands Heart Institute are Peter van Tintelen on PLN genetics, Folkert Asselbergs on epigenetics and Rudolf de Boer on clinical trials. We are also privileged to get support from Arthur Wilde (University of Amsterdam), Sakthivel Sadayappan (University of Cincinnati), and Roger Hajjar (Phospholamban Foundation), who have had a long-standing interest in cardiac physiology and pathophysiology with emphasis on underlying pathways and potential therapeutic targets. The consortium is also fortunate to embrace a patient advocate, Pieter Glijnis, incorporating the voice of the patients to research in every step. Our goal is to build and share a platform of patient data coupled with in vitro and in vivo models to promote scientific discovery and advance novel treatments. Phospholamban is a small phosphoprotein in the cardiac sarcoplasmic reticulum, and it is the major regulator of SERCA2a activity and calcium (Ca)-cycling. Chronic inhibition of SERCA2a by PLN has been implicated in the aberrant Ca-cycling of failing hearts. Studies in HF models have shown that decreasing PLN activity may rescue cardiac remodeling and dysfunction. Several human PLN mutations, leading to inhibition of Ca-uptake into the sarcoplasmic reticulum, are linked to inherited DCM.


Assuntos
Proteínas de Ligação ao Cálcio/genética , Cardiomiopatias/terapia , Fundações/organização & administração , Terapia Genética/métodos , Pesquisa Translacional Biomédica/economia , Cardiomiopatias/genética , Fundações/economia , Terapia Genética/economia , Humanos , Cooperação Internacional , Transplante de Células-Tronco/economia , Transplante de Células-Tronco/métodos , Pesquisa Translacional Biomédica/organização & administração
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