PERSPECTIVES Cochlear Gene Therapy

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.
Source: Cold Spring Harbor perspectives in medicine - Category: Research Authors: Tags: Function and Dysfunction of the Cochlea PERSPECTIVES Source Type: research

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Krystal Biotech announced Tuesday that it had named Jennifer Chien to the newly created position of chief commercial officer, effective Monday. Chien joins the Pittsburgh-based gene therapy company after serving as vice president, head of genetic diseases at Sanofi Genzyme and has more than 20 years of experience in the biopharmaceutical industry. “Jennifer’s leadership and broad experience launching new medicines will be critical as we continue to build the commercial capabilities needed…
Source: bizjournals.com Health Care:Pharmaceuticals headlines - Category: Pharmaceuticals Authors: Source Type: news
Cancer Gene Therapy, Published online: 21 January 2020; doi:10.1038/s41417-020-0162-2Prognostic role of minichromosome maintenance family in multiple myeloma
Source: Cancer Gene Therapy - Category: Cancer & Oncology Authors: Source Type: research
ConclusionsOur study demonstrates the antitumor effect of IL-24 on endometrial cancer and shows that IL-24 may be a promising therapeutic gene for endometrial cancer gene therapy.Graphical abstract
Source: Biomedicine and Pharmacotherapy - Category: Drugs & Pharmacology Source Type: research
The J.P. Morgan Healthcare conference runs every year in San Francisco, a big draw for the biotech industry, and many organizations take the opportunity to host events at the same time. Among these, the SENS Research Foundation has for the past few years hosted a pitch day in which biotech companies in the longevity industry, largely startups, present to that portion of the Bay Area investor community interested in funding the treatment of aging as a medical condition. I was there to present on progress at Repair Biotechnologies, and took some notes on the other companies as they talked about their work. Kimera Labs ...
Source: Fight Aging! - Category: Research Authors: Tags: Investment Source Type: blogs
The clustered regularly interspaced short palindromic repeats (CRISPR) and Cas9 protein system is a revolutionary tool for gene therapy. Despite promising reports of the utility of CRISPR –Cas9 for in vivo gene...
Source: Journal of Nanobiotechnology - Category: Nanotechnology Authors: Tags: Research Source Type: research
Cancer Gene Therapy, Published online: 20 January 2020; doi:10.1038/s41417-020-0160-4RNA N6-methyladenosine modification in solid tumors: new therapeutic frontiers
Source: Cancer Gene Therapy - Category: Cancer & Oncology Authors: Source Type: research
In this study, we briefly explained the molecular genetics of DMD and a historical overview of DMD gene therapy. We in particular focused on CRISPR/Cas9-mediated therapeutic approaches that used to treat DMD.
Source: Genes and Diseases - Category: Genetics & Stem Cells Source Type: research
Publication date: Available online 17 January 2020Source: European Journal of Obstetrics &Gynecology and Reproductive BiologyAuthor(s): Anna Veiga, Begoña Aran, Angel Raya, Ioannis Messinis, Tahir MahmoodAbstractOver the past 10 years’ significant research developments have taken place on human pluripotent stem cells and human embryonic stem cells to exploit the future potential in gene therapy and other focused treatments. There remains concerns around ethics of research and the fate of the human embryo used in such studies. European Board and College of Obstetrics and Gynaecology urge upon all scientists...
Source: European Journal of Obstetrics and Gynecology and Reproductive Biology - Category: OBGYN Source Type: research
Publication date: March 2020Source: Veterinary Microbiology, Volume 242Author(s): Chuangang Liu, Jianglong Li, Qili Yao, Zhisong Gao, Yanqing Cheng, Meiling Zhou, Yajie Tang, Leqiang Sun, Jinxia Dai, Gang Cao, Xiao-Lian Zhang, Ran ZhangAbstractRabies is a highly lethal infectious zoonosis caused by rabies virus (RABV), and the mortality rate is almost 100 % once clinical symptoms appear, which poses a huge threat to public health security across the many parts of the word. Vaccination is reported to be the most effective approach to prevent the disease. G protein is the only protein present on the surface of RABV, it also ...
Source: Veterinary Microbiology - Category: Veterinary Research Source Type: research
Source: Chemical and Engineering News - Category: Chemistry Authors: Source Type: research
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