Gene therapy briefs.
Ontology highlight
ABSTRACT:
SUBMITTER: Philippidis A
PROVIDER: S-EPMC4098069 | biostudies-literature | 2014 Jul
REPOSITORIES: biostudies-literature
Publications
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Project description:ContextPolicy approaches are one of the most promising population-based means of addressing the epidemic of obesity in the U.S., especially as they create supportive environments for healthy living. Policy briefs can be an effective means of disseminating research information to inform obesity prevention efforts; however, they are often ineffective because of length, density, and inaccessibility. The purposes of this project were to identify a collection of obesity-related policy briefs, analyze the content, and make recommendations for model policy briefs.Evidence acquisitionIn 2010, online searching strategies were developed with criteria that included a primary topical focus on obesity, written between 2000 and 2010, targeting any population age group, including a policy-change message, and being readily available online. The research team developed a coding tool and used it to analyze briefs. A subsample of the briefs was used for further analysis on dissemination.Evidence synthesisAnalyses were conducted on 100 briefs. Most (72%) were developed between 2005 and 2010; the average length was five pages. The majority had no tables, few figures, and only 36% included photos. The average reading level was high. A lack of monitoring or evaluating dissemination efforts prevailed.ConclusionsPolicy briefs represent an effective, often-preferred, potent tool for public health practitioners and researchers to communicate information to policymakers. Recommendations include presenting information clearly, using a concise format, including design elements, noting reference and contact information, employing active and targeted dissemination efforts, and conducting evaluation.
| S-EPMC3462489 | biostudies-literature
Project description:This article is part of a series written for people responsible for making decisions about health policies and programmes and for those who support these decision makers. Policy briefs are a relatively new approach to packaging research evidence for policymakers. The first step in a policy brief is to prioritise a policy issue. Once an issue is prioritised, the focus then turns to mobilising the full range of research evidence relevant to the various features of the issue. Drawing on available systematic reviews makes the process of mobilising evidence feasible in a way that would not otherwise be possible if individual relevant studies had to be identified and synthesised for every feature of the issue under consideration. In this article, we suggest questions that can be used to guide those preparing and using policy briefs to support evidence-informed policymaking. These are: 1. Does the policy brief address a high-priority issue and describe the relevant context of the issue being addressed? 2. Does the policy brief describe the problem, costs and consequences of options to address the problem, and the key implementation considerations? 3. Does the policy brief employ systematic and transparent methods to identify, select, and assess synthesised research evidence? 4. Does the policy brief take quality, local applicability, and equity considerations into account when discussing the synthesised research evidence? 5. Does the policy brief employ a graded-entry format? 6. Was the policy brief reviewed for both scientific quality and system relevance?
| S-EPMC3271824 | biostudies-literature
Project description:Genome editing using CRISPR-Cas systems is a promising avenue for the treatment of genetic diseases. However, cellular and humoral immunogenicity of genome editing tools, which originate from bacteria, complicates their clinical use. Here we report reduced immunogenicity (Red)(i)-variants of two clinically-relevant nucleases, SaCas9 and AsCas12a. Through MHC-associated peptide proteomics (MAPPs) analysis, we identified putative immunogenic epitopes on each nuclease. Then, we used computational modeling to rationally design these proteins to evade the immune response. SaCas9 and AsCas12a Redi variants were substantially less recognized by adaptive immune components, including reduced binding affinity to MHC molecules and attenuated generation of cytotoxic T cell responses, while maintaining wild-type levels of activity and specificity. In vivo editing of PCSK9 with SaCas9.Redi.1 was comparable in efficiency to wild-type SaCas9, but significantly reduced undesired immune responses. This demonstrates the utility of this approach in engineering proteins to evade immune detection.
2024-11-07 | PXD054579 | Pride
Project description:Over 450 million people worldwide suffer from hearing loss, leading to an estimated economic burden of ∼$750 billion. The past decade has seen significant advances in the understanding of the molecular mechanisms that contribute to hearing, and the environmental and genetic factors that can go awry and lead to hearing loss. This in turn has sparked enormous interest in developing gene therapy approaches to treat this disorder. This review documents the most recent advances in cochlear gene therapy to restore hearing loss, and will cover viral vectors and construct designs, potential routes of delivery into the inner ear, and, lastly, the most promising genes of interest.
| S-EPMC6719588 | biostudies-literature
Project description: Not available
| S-EPMC8478312 | biostudies-literature
Project description:Black six animals received either an Adeno associated virus leading to overexpression of Luciferase or Histone deacetyase 4 aminoacids 1-201. After 3 weeks of expression, animals got transaortic banding (TAC) to induce pathological cardiac remodeling. We compared both groups to Back six animals with sham surgery and overexpression of Luziferase. 3 male animals per group (5 replicates) at an age of 8 weeks were treated with an adenoassociated virus (AAV) containing a coding sequence for Luziferase or Histone Deacetylase 4 (aminoacids 1-201) und der the control of the MLC-promoter. Both groups were then exposed to transaortic constriction. As a control served AAV treated animals with overepression of luziferase and sham.
2016-07-27 | E-GEOD-72904 | biostudies-arrayexpress