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Trending again is the review article out of Wits University exploring self-amplifying #RNAs #vaccine and therapeutic candidates for infectious diseases. Join our #GTReaders here now➡️nature.com/articles/s4143… 📷: ©iStock

Online now, Huazhong University researchers use #CRISPR-based homology-directed repair of the MYBPC3 gene to rescue #HypertrophicCardiomyopathy phenotypes in an animal model. Read #GTArticle here now➡️nature.com/articles/s4143… 📷: ©iStock

Online Oxford Academic scientists University of Bristol report #genedelivery of a #longevity-associated variant of the BPIFB4 gene reverses the decline of #heart performance in #aged animal models. #GTInTheWorld 📸: ©iStock doi.org/10.1093/cvr/cv…

Trending now, Imperial College London, UCL & ImuThes Ltd scientists demonstrate long-term in vivo expression of non-viral episomal pDNA vectors containing a S/MAR attachment region and liver-specific AAT promoter. Join our #GTReaders here for more➡️nature.com/articles/gt200… 📸: ©iStock

Online now, #intrathecal injection of #AAV9-mediated HGF #genedelivery reduces astrogliosis in the motor cortex of #ALS animal models. Read #GTArticle here now➡️nature.com/articles/s4143… Work by Northwestern Helixmith Co., @SNUnow 📸: ©iStock

Trending now, #intrathecal injection of #AAV9-mediated HGF #genedelivery reduces astrogliosis in the motor cortex of #ALS animal models. For more, join our #GTReaders here now➡️nature.com/articles/s4143… Work by Northwestern Helixmith Co. & @SNUnow 📸: ©iStock

Online now, #nanoparticle mediated #genedelivery of full-length rhodopsin gene retains noncoding genomic elements and improves rod photoreceptor structural and functional recovery in animal models. #GTArticle here➡️nature.com/articles/s4143… Work by: UNC-Chapel Hill 📸: Retina Image Bank

This week, Cellusion Co. announced its #iPSC-derived #corneal endothelial #celltherapy, CLS001, has been transplanted into the first #bullouskeratopathy patient in a first-in-human trial. #GTInTheWorld 📸:©iStock

Online now the translational journey highlighting cardiac safety findings of cutting-edge #genereplacementtherapy, #Zolgensma, for #SpinalMuscularAtrophy⬇️ nature.com/articles/s4143… 📝:Novartis CHI at Crumlin UI Health and Inova researchers 📸: ©iStock #GTReview

This week the world's first vaccine, #Arexvy by GSK targeting #RespiratorySyncytialVirus for use in adults 60 and older has been approved and granted Priority Review designation by U.S. FDA 📸: ©iStock #GTInTheWorld

Trending now, intracerebral injection of #SV40 vector-mediated #genedelivery of SOD1 and GPx1 reduces #HIV gp120-induced neurotoxicity 24 weeks post-administration. For more, join #GTReaders here now➡️nature.com/articles/33030… 🧪: Thomas Jefferson University 📸: ©iStock

Reported at #EHA2023, favourable safety and efficacy #clinicaltrialresults in the first four patients with #SickleCellDisease in the RUBY trial treated with EDIT-301, the world's first CRISPR/CA12 #genetherapy by Editas Medicine. #GTIntheWorld

Online now, #AAV9 mediated #genedelivery of the SMn2B/- gene in #SMA #iPSC derived motor neurons in vitro and in a neonatal SMA mouse model restores SMN protein levels. Read #GTArticle here➡️nature.com/articles/s4143… 🧪: Rafael J. Yáñez-Muñoz's lab Royal Holloway 📸: SMA iPSC-derived MNs

Trending now is a 2004 #GTReview out of Vanderbilt University discussing the therapeutic potential of #siRNAs. For more, join our #GTReaders here now ➡️ nature.com/articles/33023… 📸: ©iStock

New issue alert ➡️nature.com/gt/volumes/30/… 📸: Horizontal brain section of an animal four weeks post intravitreal injection of #transgene #AAV2-SYN-eGFP from a paper by @BartNieuwenhu1s et al.

Bilateral intravitreal injection of triple phosphodegron mutant #AAV2 capsid is associated with increased local and systemic immune activation #OpenAccess #GTArticle ➡️nature.com/articles/s4143… 🧪: Cambridge University, Guy's and St Thomas', Moorfields Eye Hospital NHS Foundation Trust, UCL Institute of Ophthalmology & University of Melbourne 📸: Retinal wholemount

Trending now is an Orchard Therapeutics - U.S. review exploring the rationale, clinical application, technological advancement, commercialisation and challenges of targeting #hematopoieticstemcells. Join our #GTReaders here now➡️nature.com/articles/s4143… 📸:©iStock

The Clarivate 2022 Impact Factors are out, and Gene Therapy went up from 4.184 to 5.1! The editorial team couldn't be more delighted. We thank all our #GTAuthors, #GTReviewers and #GTReaders for their continued support!

Online nature, a splice switching #antisenseoligonucletode, atipeksen, administered intrathecally three years in a pediatric #AtaxiaTelangiectasia patient, was well tolerated with clinical improvements in disease progression. #GTInTheWorld 📸:©iStock nature.com/articles/s4158…

Trending now is a review discussing the outcomes of #celltherapy in #ParkinsonsDisease focusing on the types of cells and functional effects of host immune responses on graft survival and differentiation. Join our #GTReaders here now➡️nature.com/articles/s4143… 📸: ©iStock