Human Gene Therapy @HGTJournal
The first journal devoted to the science, technology and clinical application of human gene therapy. Established in 1990. Editor-in-Chief: Terry Flotte. liebertpub.com/loi/hum New Rochelle, New York Joined February 2016-
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New webinar alert - Streamlining Cell and Gene Therapy Scalability: Progress Towards a Gold Standard! Join us for this discussion with our expert panelists from across the CGT space🧬 Register today: workcast.com/register?cpak=…
"rAAV2-hSyn-hRS1, gene therapy for XLRS, specifically infects retinal ganglion cells to secrete RS1 protein." Read more in this new paper: liebertpub.com/doi/10.1089/hu…
Learn more about an ELISpot assay to examine AAV8 and AAV9 T cell-dependent responses to inform recombinant AAV vector design: liebertpub.com/doi/10.1089/hu…
Exciting news: AAV9-therapy shows life-saving potential in a model of myasthenia with episodic apnea! A breakthrough in genetic medicine: liebertpub.com/doi/10.1089/hu…
Unlocking precise AAV antibody detection with CRISPR-Cas9: Introducing AAV-HDR Method. Elevate your research! #CRISPR #AAV #AntibodyDetection liebertpub.com/doi/10.1089/hu…
Tagged IDS corrects peripheral tissues, but not cartilage during gene therapy for MPS II: liebertpub.com/doi/10.1089/hu… #genetherapy
New review on the latest in gene therapy for organ transplantation and progress toward clinical translation: liebertpub.com/doi/10.1089/hu… #genetherapy #organtransplantation
Tired of endless optimisation? In a new manuscript, Whittaker et al. show that Response Surface Methodology allowed fast, parsimonious and definitive preclinical optimisation of a new gene therapy for p67-deficient CGD. liebertpub.com/doi/10.1089/hu…
Ultragenyx’s Gene Therapy Ameliorates Pediatric Neurodegenerative Disorder. Learn more: ow.ly/8TTh50QyLKn
Our UC Davis Vector Core is a centralized service for development and production of viral vectors necessary for gene transfer in research experiments and pre-clinical studies. Expert staff enable the design and manufacture of novel vectors & purified recombinant retrovirus and…
CASGEVY Makes History as @US_FDA Approves First #CRISPR/Cas9 Genome Edited Therapy. Details in #GeneTherapy Briefs in @HGTJournal: liebertpub.com/doi/10.1089/hu…
Forge Biologics shares their novel finding that redefines the requirements of #AAV production in HEK293 cells! Discover more here: liebertpub.com/doi/10.1089/hu…
Highlighting ethical, legal, and social implications, and the multi-party role for human gene and cell therapy. liebertpub.com/doi/10.1089/hu…
Activist Investor Reported to Take $1 Billion Stake in @BioMarin. Details in #GeneTherapy Briefs in @HGTJournal: liebertpub.com/doi/10.1089/hu…
This editorial comments on the Lived Experience of families facing tough decisions about whether and when to undergo gene therapy. liebertpub.com/doi/10.1089/hu… #genetherapy #humangenetherapy
Scoping review findings indicate that research on the "lived experience" of participating in GT trials is seriously lacking. Our goal is to design more patient-centered trials and supportive systems for trial participants and their families. liebertpub.com/doi/10.1089/hu…
Gene transfer based on AAV vectors can potentially cure genetic diseases but commonly require doses that can cause adverse events. liebertpub.com/doi/10.1089/hu…
"Genome Editing in Engineered T Cells for Cancer Immunotherapy" has been made temporarily #FreeAccess through December 31, 2023. liebertpub.com/doi/full/10.10…
"Genome Editing in Engineered T Cells for Cancer Immunotherapy" has been made temporarily #FreeAccess through December 31, 2023. liebertpub.com/doi/full/10.10…
First Prime Editing Clinical Trial Expected in 2024. Details in #GeneTherapy Briefs in @HGTJournal: liebertpub.com/doi/full/10.10…
A new LC-MS/MS method to measure residual Tat protein in lentiviral vectors for #genetherapy has been developed. It is validated, reliable and precise. The LC-MS/MS platform can be used for other residual proteins. #lentivirus #massspectrometry liebertpub.com/doi/10.1089/hu…
ASGCT @ASGCTherapy
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7February: Today marks the start of my 13th year at @LiebertPub, where I report and write and podcast for @GENbio, @GENbiotechJrnl, and @HGTJournal. Thanks to @KPhilippidis & my colleagues for all their help during all that time. Looking forward to the next several years...
We have exciting news to share: a pair of significant GTP manuscripts were published in @NatureBiotech & @HGTJournal! The findings from these studies carry important implications for the safety profile & long-term efficacy of AAV-based gene therapies: pennmedicine.org/news/news-rele…
Webinar: Design of Experiment Based Optimization of Full AAV Particle Enrichment Using Stunner® as a High-Throughput Analysis Tool. @unchainedlabs #AAV #capsid #webinar ow.ly/oKjc50Q1SN9
Webinar: Design of Experiment Based Optimization of Full AAV Particle Enrichment Using Stunner® as a High-Throughput Analysis Tool. @unchainedlabs #AAV #capsid #webinar ow.ly/wRwo50Q17QU
Call for Papers! Application of #MachineLearning and Novel #GenomeSequencing Technology in #GeneTherapy Special Issue @HGTJournal will focus on innovative deep learning models as an emerging approach in genome sequencing for use in gene therapy. SUBMIT: ow.ly/Y6Ac50Puc2s
Special Issue: #GeneTherapy for #MuscleDisorders In this special issue of @HGTJournal, experts in the field present reviews on some of the most pressing issues and recent advances in the field. Read the full issue: ow.ly/HkVi50OV55B
Happy 20th anniversary @_BSGCT! Here's to many more! 🎂🥂🍾🎉
Anticipating #ASGCT23 with the Editor-in-Chief of @HGTJournal Terry Flotte. Next week, Los Angeles hosts what is likely to be the largest #genetherapy conference in history as the #ASGCT arrives. Read more: ow.ly/HNoh50OnX0S
Analytical Methods for Preclinical Stage #GeneTherapy Programs Read the full study "Application of Size Exclusion Chromatography with Multiangle Light Scattering in the Analytical Development of a Preclinical Stage Gene Therapy Program" in @HGTJournal: ow.ly/9p7N50OkmE9
Study @HGTJournal reports findings of 6 #AAV vectors in 12 preclinical models of the human #liver, aiming to uncover which combination is most relevant for identification of AAV capsid variant for safe & efficient #genetherapy to primary human hepatocytes: ow.ly/bt8p50Oalx3
Congratulations to @AWesthaus and CMRI’s Translational Vectorology team for their publication in @HGTJournal which was described as a “highly innovative’’ approach to studying the safest delivery of gene therapy to the human liver.
Where Does #GeneTherapy Go After #CerebrospinalFluidDelivery? A new report in @HGTJournal presents a literature review of preclinical studies of adeno-associated virus (#AAV)-based gene therapy biodistribution following cerebrospinal fluid delivery: ow.ly/SN9b50N4egp
An interesting article published in @HGTJournal that highlights public perceptions of the ethics of #genetherapy has been cited in @sciam. Read it here: ow.ly/cI7L50GR290
New Advances in #HumanGeneTherapy Read new articles to explore the latest research and discoveries in the field of human gene therapy -- Access here: ow.ly/BGUR50GnNFL ow.ly/IZeB50GnNFK ow.ly/OblN50GnNFM #GeneTherapy #GeneDelivery #AAV @HGTJournal
Human Gene Therapy announces a new impact factor of 5.695*, a 26% increase over last year! Ensure you have access to all of the content in @HGTJournal in the coming year – recommend a subscription to your library today: ow.ly/JNay50Fqj1f #GeneTherapy #HumanGeneTherapy
Ly6a Differential Expression in Blood–Brain Barrier Is Responsible for Strain Specific Central Nervous System Transduction Profile of AAV-PHP.B Read this top-cited article in @HGTJournal today: ow.ly/7dQu50F1oUs
@KevinADavies Thank you, Great talks! I enjoy all talks, specially #MatthewPorteus talk!
Amazing news. Emily was the 1st pediatric patient to receive a CAR-T and her survival was a catalyst for a step change in medicine, with 2 CAR-Ts now approved and many more in the pipeline. She was 6 yrs old with leukaemia & had over 16 months of chemo before the treatment 🙏
Today, Emily is 8 years cancer free! #WeBelieve #ActivateTheCure
Dr. Ed Rebar of @SangamoTx shares exciting genomic therapies in development, overcoming lab challenges early in his career, the thrill of being at meetings when new discoveries are announced, traditions that bring their team together, & more! peoplebehindthescience.com/dr-edward-reba… #GeneEditing
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