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🎉bioRxiv & medRxiv are now managed by openRxiv, a new independent nonprofit. This transition allows for more flexibility and innovation. Our community-centered mission remains being a free service to authors ensuring rapid sharing of scientific information before peer review. 🧵

AAVrh10 gene therapy shows promise for CMT1X. Intrathecal (spine) delivery restores Cx32 in Schwann cells, improving motor function in mice with minimal side effects. A step closer to treating this neuropathy? 🧬 Read more: buff.ly/qk4ukKn #GeneTherapy #CMT1X

Debate in The Lancet on nAMD outcomes of AbbVie and RegenxBio's AAV gene therapy RGX-314: 14-letter BCVA gain in some, but critics question CRT stability. Authors cite prolonged exudation control, yet dose-related risks like pigmentary changes persist. #AMD $ABBV $RGNX AbbVie

HSV-1 vectors show promise in gene therapy for cancer, vaccines, & neurological diseases. Learn how they can deliver genes effectively & safely. Free PDF: buff.ly/Tk89iCL

Today at Cell Systems we report in back-to-back papers (!) led by Nat B. Wang 🧬🧑🏻‍🔬🏳️‍🌈 on how cell state potentiates the influence of individual transcription factors to drive cell-fate programming & how we used these insights to scale up production of motor neurons from primary

⚡ Breakthrough Study Doshi et al. reveal CD19-targeted CAR-T cells eradicate pre-existing AAV neutralizing antibodies (NAb) in mice, enabling successful systemic redosing of AAV8 vectors. This tackles a major hurdle in AAV gene therapies, where NAb often block efficacy due to

Study on cancer survivors using oral doses of either dasatinib and quercetin, or fisetin or other senescent cell removers to extend health span. pulse.ly/roua1qswpy

🚀 Miniaturizing base editors for gene therapy! Our new Ambryon Insights post "Making Base Editors Smaller for Gene Therapy," dives into how a smaller Cas9 orthologs enhance base editing efficiency in human cells and their significance in advancing gene therapy. Inspired by

Gene therapy for cystic fibrosis shows promise. Learn about CFTR delivery, CRISPR/Cas9, & challenges in this new review paper. Free PDF: buff.ly/hEYgi4Q #CysticFibrosis #GeneTherapy #CRISPR

🚀 Ultragenyx's AAV8 gene therapy DTX401 shows promise for glycogen storage disease Type Ia (GSDIa). A phase 1/2 trial reduced cornstarch needs by 68% in adults, improving glycemic control with a safe profile. $RARE #GeneTherapy #AAV onlinelibrary.wiley.com/doi/10.1002/ji…

⚡ Breakthrough Study A new Nature Communications study shows MyoAAV vectors deliver CRISPR-Cas9 to restore dystrophin in Duchenne muscular dystrophy (DMD) mice, achieving widespread muscle repair with minimal off-target effects. First described in 2021, MyoAAV is a family of

A new Adv. Sci. study demonstrates that a single AAV-ie-Eh3 injection rescues hearing and vestibular function in mice with Atp6v1b2 knockout, linked to syndromes like DDOD. This congenital deafness model shows hair cell loss due to lysosomal dysfunction, which the therapy

New study demonstrates that AAV9-mediated delivery of GBA1 and GDNF rescues neurological defects in a murine model of neuronopathic Gaucher disease (nGD). Gaucher disease, caused by GBA1 mutations, leads to glucocerebrosidase deficiency, causing glucosylceramide accumulation,

AAV9 delivery of Magea13 attenuates myocardial injury in mouse model of acute myocardial infarction (AMI) by inhibiting the cAMP-PKA signaling pathway. AMI, caused by prolonged coronary artery occlusion, leads to cardiac tissue damage and heart failure; Magea13 overexpression in

⚡ Breakthrough Study A new study in Nat. Commun. unveils berberine-inspired ionizable lipid nanoparticles (LNPs) designed to deliver nucleic acid therapeutics across the blood-brain barrier (BBB). Leveraging the structure of protoberberine alkaloids, these LNPs enhance