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Therapy: Gene Therapy
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Total 5568 results found since Jan 2013.
Simple chitin-based cell culture platform for production of biopharmaceuticals
CONCLUSIONS: Cellhesion® VP provides a simple and streamlined expansion culture system for the production of biopharmaceuticals.PMID:37606752 | DOI:10.1007/s10529-023-03422-7
Source: Biotechnology Letters - August 22, 2023 Category: Biotechnology Authors: Katsuhiko Kida Daisuke Hatanaka Masataka Minami Taiyo Suzuki Masashi Iwakami Masaki Kobayashi Hisato Hayashi Hiroharu Kawahara Masato Horikawa Tatsuro Kanaki Source Type: research
Soluble CX3CL1-expressing retinal pigment epithelium cells protect rod photoreceptors in a mouse model of retinitis pigmentosa
CONCLUSIONS: Transgenic hESCs differentiate into hRPE cells and retain sCX3CL1 transgene expression both in vitro and in vivo. Moreover, hRPE cells delivered to the subretinal space of rd10 mice prevented photoreceptor degeneration in a local-acting manner, suggesting that this approach could have applications for preserving photoreceptors in specific subregions of the retina, such as the macula. Overall, our study not only reveals the potential of a combined cell and gene therapy for the treatment of RP, but also the possibility of using hRPE cells to deliver therapeutic biologics in situ to treat diseases over long-term....
Source: Cell Research - August 22, 2023 Category: Cytology Authors: Eric D Jong Sabiha Hacibekiroglu Lily Guo Evan Sawula Biao Li Chengjin Li Margaret T Ho Molly S Shoichet Valerie A Wallace Andras Nagy Source Type: research
Simple chitin-based cell culture platform for production of biopharmaceuticals
CONCLUSIONS: Cellhesion® VP provides a simple and streamlined expansion culture system for the production of biopharmaceuticals.PMID:37606752 | DOI:10.1007/s10529-023-03422-7
Source: Biotechnology Letters - August 22, 2023 Category: Biotechnology Authors: Katsuhiko Kida Daisuke Hatanaka Masataka Minami Taiyo Suzuki Masashi Iwakami Masaki Kobayashi Hisato Hayashi Hiroharu Kawahara Masato Horikawa Tatsuro Kanaki Source Type: research
Improved Cardiac Function in Postischemic Rats Using an Optimized Cardiac Reprogramming Cocktail Delivered in a Single Novel Adeno-Associated Virus
CONCLUSIONS: Collectively, these results advance the application of cardiac reprogramming gene therapy as a viable therapeutic approach to treat chronic heart failure resulting from ischemic injury.PMID:37602409 | DOI:10.1161/CIRCULATIONAHA.122.061542
Source: Circulation - August 21, 2023 Category: Cardiology Authors: Huanyu Zhou Jin Yang Chetan Srinath Aliya Zeng Iris Wu Elena C Leon Tawny Neal Qureshi Christopher A Reid Emily R Nettesheim Emma Xu Zoe Duclos Tamer M A Mohamed Farshad Farshidfar Anthony Fejes Jun Liu Samantha Jones Charles Feathers Tae Won Chung Frank Source Type: research
Herpes simplex virus gene therapy for dystrophic epidermolysis bullosa (DEB)
Cell. 2023 Aug 17;186(17):3523-3523.e1. doi: 10.1016/j.cell.2023.07.031.ABSTRACTThe FDA has recently approved Krystal biotech's beremagene geperpavec (B-VEC, Vyjuvek) to treat the wounds of dystrophic epidermolysis bullosa (DEB) patients. This represents a giant step, not only toward the treatment of this devastating disease, but also for the whole field of non-replicative (nr) recombinant HSV-1 vectors for gene therapy. To view this Bench to Bedside, open or download the PDF.PMID:37595560 | DOI:10.1016/j.cell.2023.07.031
Source: Herpes - August 18, 2023 Category: Infectious Diseases Authors: Alberto L Epstein Cornelia Haag-Molkenteller Source Type: research
Successful Transduction of Target Gene Mediated by Adeno-Associated Virus 2 into Lens Epithelial Cells in Rats
In this study, we compared the efficiency of intravitreal injection of six AAV serotypes (AAV2, AAV5, AAV6, AAV8, AAV9, and AAVDJ) to transduce lens and retina in rats, The expression and localization of the reporter gene ZsGreen in the lens and retina were examined using immunofluorescence staining, and the relative expression of ZsGreen mRNA was detected using RT-qPCR. Our results demonstrated that AAV2 had the highest efficiency in transducing LECs. All six AAV serotypes could transduce the retina. To validate this observation, we further constructed an AAV2 vector with exogenous gene senescence marker protein 30 (SMP30...
Source: Journal of Virological Methods - August 17, 2023 Category: Virology Authors: Yongshun Liang Tian Lan Qingqiao Gan Hao Liang Source Type: research
