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- New on FirstView: "Balancing Progress and Precautions with Emerging Biotechnologies: Evaluating the Process of Gene Therapy Research Becoming'Normal'” by Stephanie Solomon Cargill. Many thanks to @nadagligorov.bsky.social for assistance. #GeneTherapy www.cambridge.org/core/journal... #GeneTherapy
- ⚡Our January issue is live! Including the first Nature Medicine Commission Report (introduced in our Editorial), new research on #infertility, #huntingtonsdisease, #cancer #cachexia, #HIV, #genetherapy, #microbiome therapy, News & Opinion and much more nature.com/nm/volumes/32/issues/1
- Larry Luxner, senior correspondent, interviews Barry Byrne, MD, PhD, an expert on #PompeDisease, director of the @ufresearch.bsky.social Powell Gene Therapy Center, and Chief Medical Advisor to the @mda.org 🎙️ Listen to the podcast here: bit.ly/494CQlK #RareDisease #MedSky #GeneTherapy
- A new uniQure study shows promise for improving AAV-based #genetherapy delivery to the CNS. The team used the LifeCanvas pipeline to visualize biodistribution throughout whole mouse brains. 🐭 🧠 Learn more & see their #SmartSPIM data: bit.ly/4rixY3v
- A landmark clinical trial of gene therapy infusion for hemophilia B revealed surprising findings—reshaping the future of genetic medicine. Read the full Turning Point essay in Nature Medicine: ow.ly/RVST50XvVs0 #GeneTherapy #HemophiliaB #NatureMedicine
- ⚡Our November issue is out! Featuring trials of #CARTcells for #LUPUS, an #ADC for #lungcancer, cardiac #genetherapy, an #ebola #vaccine, discovery of microbial elements in #braintumors, #liquidbiopsies, #newbornscreening, News, Reviews, & more. nature.com/nm/volumes/31/issues/11
- Jennifer E. Adair, PhD, will use a $1.27M National Institutes of Health Fogarty International Center Global Infectious Disease research grant to expand the global cell and gene therapy network and build capacity for an international response to the HIV epidemic: direc.to/nH78 #GeneTherapy #HIV
- New method ➡️ Domain-substituted IGF2 tag modulates targeting of lentiviral #GeneTherapy for #HunterSyndrome By F. Catalano, WWMP Pijnappel & colleagues @erasmusmc.bsky.social 🗞️ doi.org/10.1038/s443...
- 💡A new RNAi strategy described in NARMME embeds miR-based shRNAs into a gamma-retroviral intron, enabling gene knockdown and protein coexpression with high viral titers. 📖 doi.org/10.1093/narm... #RNAi #GeneTherapy #RetroviralVectors #MolecularBiology #NARMME #OpenAccessPublication
- 🚨Just published - Multiplex engineering using microRNA-mediated gene silencing in CAR T cells #celltherapy #genetherapy #immunotherapy www.frontiersin.org/journals/imm...
- A clinical study of gene therapy for GM2 gangliosidosis, published today in Nature Medicine, shows encouraging results at UMass Chan Medical School: direc.to/nxxq “Biochemically it worked.” - Heather Gray-Edwards, DVM, PhD #GeneTherapy #TaySachs #Sandhoff @natmed.nature.com @uofmass.bsky.social
- 🚨 #PublicationAlert Gene therapy for pediatric vision loss — powered by human retina models! Our novel #RetinaOnChip mimics the human #Retina using #Organoids to model #CLN2 disease and test #GeneTherapy — now in a first-in-human trial! 📖 DOI: doi.org/10.1016/j.xc...
- 🎧 Don’t miss this podcast with Dr. Tippi MacKenzie, Broad Center Director, on in utero therapies & the future of regenerative medicine. 📍19:40: vision for the Center → discover, engineer, heal. 🔗 bit.ly/46qjYgi #UCSF #StemCells #FetalSurgery #GeneTherapy #GenomeEditing #DiscoverEngineerHeal
- When UCLA's Dr. Gerald Lipshutz heard parents' raw accounts of children with creatine transporter deficiency—unable to speak, suffering seizures—he was moved to action. He's now developing a one-time #GeneTherapy that could transform these families' lives. ➡️ bit.ly/44P1rrp
- 🧪In May 2025, the world’s first patient, a baby with a rare metabolic disorder, was treated with CRISPR gene therapy at Children’s Hospital of Philadelphia and Univ. of Penn. Is this a major breakthrough, bioethics challenge, or both? 🧬🔬 #SciComms #CRISPR #GeneTherapy #RareDisease #Genomics.
- Hear how gene therapies can address serious genetic diseases before birth. Dr. Tippi MacKenzie will deliver a George Stamatoyannopoulos Memorial Lecture at the @asgct.bsky.social Annual Meeting on May 15. #ASGCT2025 #GeneTherapy #PrenatalResearch #raredisease
- 🔥 Breaking research! Fresh off Metabolic Engineering, Leopold Zehetner reveals how to boost AAV production in HEK293 for gene therapy. 🚀 Live in 20 min! Don't miss his deep dive into #MetabolicModeling in biotech! 🌍💡 🗓️ Today | 🕒 13:30 CET 🔗 Join now: lnkd.in/dFfRRs9W #Bioengineering #GeneTherapy
