Human Gene Therapy

Join our webinar on 11/13 to explore iPSC-derived assays in gene therapy: assay design, quantifiable potency, and regulatory insights. Register now: hubs.li/Q03MNBCL0 #GeneTherapy #iPSC #Webinar #Biotech #Ncardia

FREE WEBINAR! Advancing Gene Therapy Potency Testing with iPSC-Based Systems. REGISTER: hubs.li/Q03MNBCM0 #Ncardia #genetherapy #assays #therapyvectors #freewebinar

NEW WEBINAR! On 11/13, our experts discuss strategies for implementing iPSC-derived assays in gene therapy development pipelines, with a focus on assay design, quantifiable potency metrics, and considerations for regulatory alignment. REGISTER: hubs.li/Q03MNtlw0 #Ncardia

Join GEN for The State of CRISPR and Genome Editing virtual summit on June 11! Hear from experts like Feng Zhang and Fyodor Urnov. Don't miss out—register for free today! hubs.li/Q03q4VKD0

Are you in New Orleans for the 2025 ASGCT Annual Meeting? Stop by Booth #1350 to catch up with the Mary Ann Liebert, GEN, and Inside Precision Medicine teams! Pick up your copies of Human Gene Therapy, GEN, CRISPR Journal, and more at this year's meeting. #ASGCT2025

Read the April issue of Human Gene Therapy with your free digital edition, available now during the @ASGCTherapy Annual Meeting: engraffo.com/p/35fKnSR01F

Read the April issue of Human Gene Therapy with your free digital edition, available now during the @ASGCTherapy Annual Meeting: engraffo.com/p/35fKnSR01F

Online now: Creating New Cis-Regulatory Elements of HBD to Reactivate Delta-Globin: liebertpub.com/doi/10.1089/hu…

Online now: @BeamTx Results Show First Genetic Correction of Disease-Causing Mutation: liebertpub.com/doi/10.1089/hu…

Online now: Meta-Analysis and Optimization of the In Vitro Immortalization Assay for Safety Assessment of Retroviral Vectors in #GeneTherapy: liebertpub.com/doi/10.1089/hu…

Tomorrow -- @GENbio Webinar: Making Protein Therapeutics Affordable and Accessible Using Fully-Connected Continuous Biomanufacturing. 8:00 am PT, 11:00 am ET, 17:00 CEST. Register free now at: genengnews.com/multimedia/web…

Identifying a Novel Factor in Canavan Disease Pathogenesis -- Read the Open Access article now: liebertpub.com/doi/10.1089/hu…

Prediction of Adeno-Associated Virus Fitness with a Protein Language-Based Machine Learning Model... Read the Free Access research article by Christian Mueller and coauthors from @sanofi : liebertpub.com/doi/10.1089/hu…

Machine Learning Model to Predict the Fitness of AAV Capsids for #GeneTherapy. Read the Free Access research article by Christian Mueller and coauthors from @sanofi : liebertpub.com/doi/10.1089/hu…

Prediction of Adeno-Associated Virus Fitness with a Protein Language-Based Machine Learning Model... Read the Free Access research article by Christian Mueller and coauthors from @sanofi : liebertpub.com/doi/10.1089/hu…

Machine Learning Model to Predict the Fitness of AAV Capsids for #GeneTherapy. Read the Free Access research article by Christian Mueller and coauthors from @sanofi : liebertpub.com/doi/10.1089/hu…

New articles from the February Issue, free for a limited time -- Directed Evolution of AAV9 for Efficient Gene Expression in Cardiomyocytes In Vitro and In Vivo: liebertpub.com/doi/10.1089/hu…

New articles from the February Issue, free for a limited time -- Current and Emerging Issues in Adeno-Associated Virus Vector-Mediated Liver-Directed #GeneTherapy: liebertpub.com/doi/10.1089/hu…

New articles from the February Issue, free for a limited time -- Thrombotic Microangiopathy Associated with Systemic Adeno-Associated Virus Gene Transfer: Review of Reported Cases. liebertpub.com/doi/10.1089/hu…

Call for Papers -- Special Issue on #GeneTherapy for Diseases of the Respiratory System. Deadline for Manuscript Submissions: April 25, 2025. Submit Today! home.liebertpub.com/cfp/special-is…