Latest posts 5
Just out: a comprehensive review of gene therapies in ALS β covering neuroprotection, ASOs, RNAi, CRISPR etc.; includes tables summarizing preclinical & clinical data, discusses hurdles & latest trial outcomes.
πRead more: liebertpub.com/doi/full/10.11β¦
πRead more: liebertpub.com/doi/full/10.11β¦
π¬π§¬ Our latest paper has just been published in Molecular Therapy (@MolTherapy)! In this study, conducted at @INC_UAB @UABBarcelona , we developed an intravenous gene therapy for MLC, an ultrarare leukodystrophy.
cell.com/molecular-therβ¦
www.cell.com Gene therapy rescues brain edema and motor function in a mouse model of megalencephalic leukoence... Bosch and colleagues demonstrate that astrocyte-specific AAV gene therapy restores MLC1 expression, reverses white matter vacuolation, and improves motor function in Mlc1β/β mice for up to 1 year.... cell.com/molecular-therβ¦
Our PI participated in the 9th Annual Wolfram Syndrome Research Symposium where she gave a talk on our latest findings. Thank you to the Snow Foundation for organizing this impactful event. We appreciate the opportunity to engage with the community!
π shorturl.at/yci0p
π shorturl.at/yci0p
Last week at #ESGCT2024 was incredible! Our team, alongside the Gene Therapy for CNS lab, showcased our work with 3 posters on gene therapies for rare diseases: MLC, SPG52, and ALS. It's always exciting to contribute to the future of the field and connect with colleagues!