Dataset Information

Taking Stock of Retinal Gene Therapy: Looking Back and Moving Forward.

ABSTRACT: Over the past 20 years, there has been tremendous progress in retinal gene therapy. The safety and efficacy results in one early-onset severe blinding disease may lead to the first gene therapy drug approval in the United States. Here, we review how far the field has come over the past two decades and speculate on the directions that the field will take in the future.

SUBMITTER: Bennett J

PROVIDER: S-EPMC5417792 | biostudies-literature | 2017 May

REPOSITORIES: biostudies-literature

ACCESS DATA

Publications

Taking Stock of Retinal Gene Therapy: Looking Back and Moving Forward.

Bennett Jean J

Molecular therapy : the journal of the American Society of Gene Therapy 20170405 5

Over the past 20 years, there has been tremendous progress in retinal gene therapy. The safety and efficacy results in one early-onset severe blinding disease may lead to the first gene therapy drug approval in the United States. Here, we review how far the field has come over the past two decades and speculate on the directions that the field will take in the future. ...[more]

PMID: 28391961

Similar Datasets

Project description:The development of treatment biomarkers for psychiatric disorders has been challenging, particularly for heterogeneous neurodevelopmental conditions such as attention-deficit/hyperactivity disorder (ADHD). Promising findings are also rarely translated into clinical practice, especially with regard to treatment decisions and development of novel treatments. Despite this slow progress, the available neuroimaging, electrophysiological (EEG) and genetic literature provides a solid foundation for biomarker discovery. This article gives an updated review of promising treatment biomarkers for ADHD which may enhance personalized medicine and novel treatment development. The available literature points to promising pre-treatment profiles predicting efficacy of various pharmacological and non-pharmacological treatments for ADHD. These candidate predictive biomarkers, particularly those based on low-cost and non-invasive EEG assessments, show promise for the future stratification of patients to specific treatments. Studies with repeated biomarker assessments further show that different treatments produce distinct changes in brain profiles, which track treatment-related clinical improvements. These candidate monitoring/response biomarkers may aid future monitoring of treatment effects and point to mechanistic targets for novel treatments, such as neurotherapies. Nevertheless, existing research does not support any immediate clinical applications of treatment biomarkers for ADHD. Key barriers are the paucity of replications and external validations, the use of small and homogeneous samples of predominantly White children, and practical limitations, including the cost and technical requirements of biomarker assessments and their unknown feasibility and acceptability for people with ADHD. We conclude with a discussion of future directions and methodological changes to promote clinical translation and enhance personalized treatment decisions for diverse groups of individuals with ADHD.

Project description:BACKGROUND:Ethics teaching is globally considered an essential part of medical education fostering professionalism. It does not only provide knowledge for good clinical conduct, but also trains medical students as virtuous practitioners. Although Turkey has had a considerable experience in ethics education of healthcare professionals, the general state of ethics curricula at medical schools in Turkey is unknown. METHODS:The purpose of this study was to collect comprehensive data about the ethics education programs at medical schools in Turkey. To this aim, we designed a cross-sectional descriptive questionnaire survey which focuses on the content, teaching years, teaching, assessment and evaluation methodologies, workforce and infrastructure. We delivered the questionnaire to all medical schools in Turkey. Seventy-nine medical schools participated in this study (response rate: 78%). RESULTS:Although most institutions had an undergraduate ethics curriculum (91.1%), the findings suggest deficiency of teaching personnel (34.2% had no instructors). Furthermore, the distribution and composition of the workforce was imbalanced. The content varies largely among institutions. Medical schools with an ethics department were more likely to diversify teaching topics. However, ethics education was largely based on the four-principle approach. The content was usually conveyed to students theoretically. Around 90% of schools had classroom lectures. It is the only method used at one-third of them. Clinical ethics education was mostly lacking. Multiple-choice tests were widely used to assess and evaluate student attainments (86.1%). CONCLUSIONS:Staff qualified to teach ethics and ethics education integrated into the six-year medical curriculum given by a multidisciplinary team are urgent necessities. Considering teaching, assessment and evaluation methodologies used, most medical schools seem to fall short of fostering students to develop ethical attitudes. Endeavors aiming for modern topics should be encouraged. As the organization ethics education change continuously, we think that a platform for monitoring ethics education at medical schools in Turkey should be established. Such a body would help ethics instructors to network and find solutions to current problems and build shared wisdom.

Dataset Information

Taking Stock of Retinal Gene Therapy: Looking Back and Moving Forward.

Publications

Taking Stock of Retinal Gene Therapy: Looking Back and Moving Forward.

Similar Datasets

OmicsDI is part of the ELIXIR infrastructure

Tweets