Viral gene therapy for paediatric neurological diseases: progress to clinical reality onlinelibrary.wiley.com/doi/10.1111/dm… Congrats! @RickPrivolizzi Hester Chu Maha Tijani 👏👍🙏Bernard Dan UCL EGA Institute for Women's Health

Thanks to the patients and their families who participated in our study, and our collaborators around the globe. Genetic cause of X-linked intellectual disability - due to mutations in WNK3 kinase! We cloned it in 2003. Never imagined it would be human disease gene!

AAVs for microglia 🔥 👇 Directed evolution of adeno-associated virus for efficient gene delivery to microglia | Nature Methods nature.com/articles/s4159…

Congrats to @MGH_CGM faculty members, Florian Eichler and @GeneStrokeLab, and their team on the FDA approval of gene therapy for early active cerebral ALD MassGeneral News @MGH_RI MGH Neurology usnews.com/news/top-news/…

Congratulations Patty Musolino @GeneStrokeLab & David Chung David Chung on receiving an NINDS R01 to develop gene therapy for Smooth Muscle Dysfunction Syndrome, a rare disease caused by a mutation in the ACTA2 gene. MGH Neurology MassGeneralNeuroscience

Editing a gateway for #CellTherapy across #BloodBrainBarrier An excellent review🤠 Learn a lot! Lesson from Leukocyte-#BBB interaction-> Engineering strategies->⏫Cell delivery to injured🧠#Stroke Beatriz Achon Buil Christian Tackenberg Ruslan Rust 🧬🧠 Brain 2022 academic.oup.com/brain/advance-…

Researchers examine current knowledge on GABAergic and glutamatergic synapse responses to perturbances in activity, with a focus on postsynaptic plasticity induced by short-term pharmacological treatments. Full study in Frontiers in Synaptic Neuroscience Frontiers - Neuroscience⬇️

Today's FDA approval of #Trofinetide could bring new, much needed hope to many with #Rettsyndrome and their families. In 2009 Sur Lab made the first preclinical showing that IGF-1 could have therapeutic potential. picower.mit.edu/news/3-questio… @mit @mitscience MIT Brain and Cognitive Sciences

Congratulations to scientists from the Moss Laboratories, whose research identified a new potential treatment for drug-resistant epilepsy! news-medical.net/news/20230315/…

#PatriciaMusolino #FlorianEichler & colleagues at @GeneStrokeLab MGH Neurology MGH Martinos Center CGM at MGH Mass General Imaging report a breakthrough in gene therapy to improve brain microvascular function: nature.com/articles/s4146… @MGH_RI

Next generation of mouse models has arrived. Congrats to all authors! Jacks Lab David R. Liu nature.com/articles/s4158…

At the MGH Myhre Syndrome Clinic, in addition to caring for people, they conduct research to better understand the complex problems of Myhre Syndrome. The clinic is run by by Angela Lin, MD and @MarkELindsay! Read more: mgriblog.org/2023/06/26/thr… #benchtobedside

Folks, I give you ... a Kaplan-Meier curve to live for courtesy of Dr Patricia Musolino and colleagues at MGH. This is viral gene therapy for a devastating disease in a mouse model. I am excited for these data (abstract #35 #ASGCT2024) and a wealth more.

Mass General Neurologist Patricia Musolino, MD, PhD, (@GeneStrokeLab), is leading a preclinical and clinical research collaboration working towards developing advanced #GeneTherapy technologies for the treatment of rare cerebrovascular diseases. spklr.io/6014oI48

Delighted to see our CLN1 & CLN2 Enteric Nervous System gene therapy paper published in Science Translational Medicine today! Big team effort with many great colleagues Heuckeroth Lab WashU Medicine Pediatrics & directly inspired by Batten families BDSRA Foundation NINDS Tracy VanHoutan science.org/doi/10.1126/sc…

Proud to be here today. Thank you Bapa for making me what I am today! Wishing you good health and prosperity.

In pictures: How #CRISPR, TALENs and ZFNs work #OutlookGeneEdit #GeneEditSummit nature.com/nature/journal…