Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

Jerry R. Mendell, Samiah Al-Zaidy, Richard Shell, +23

New England Journal of Medicine

Direct conversion of patient fibroblasts demonstrates non-cell autonomous toxicity of astrocytes to motor neurons in familial and sporadic ALS

Kathrin Meyer, Laura Ferraiuolo, Carlos J. Miranda, +12

Proceedings of the National Academy of Sciences

Improving Single Injection CSF Delivery of AAV9-mediated Gene Therapy for SMA: A Dose–response Study in Mice and Nonhuman Primates

Kathrin Meyer, Laura Ferraiuolo, Leah Schmelzer, +11

Molecular Therapy

Oligodendrocytes contribute to motor neuron death in ALS via SOD1-dependent mechanism

Laura Ferraiuolo, Kathrin Meyer, Thomas W. Sherwood, +11

Proceedings of the National Academy of Sciences

Major histocompatibility complex class I molecules protect motor neurons from astrocyte-induced toxicity in amyotrophic lateral sclerosis

SungWon Song, Carlos J. Miranda, Lyndsey Braun, +8

Nature Medicine

Amyotrophic Lateral SclerosisMedicineProtect Motor NeuronsNeuroprotectionNeurology +7

Delayed Disease Onset and Extended Survival in the SOD1^G93ARat Model of Amyotrophic Lateral Sclerosis after Suppression of Mutant SOD1 in the Motor Cortex

Gretchen M. Thomsen, Genevíève Gowing, Jessica Latter, +9

Journal of Neuroscience

Gene Therapy Corrects Brain and Behavioral Pathologies in CLN6-Batten Disease

Jacob T. Cain, Shibi Likhite, Katherine A. White, +14

Molecular Therapy

Optimization and validation of CAR transduction into human primary NK cells using CRISPR and AAV

Meisam Naeimi Kararoudi, Shibi Likhite, Ezgi Elmas, +12

Cell Reports Methods

Systems BiologyAav VectorsEngineeringGenome SurgeryCar Transduction +12

Translating SOD1 Gene Silencing toward the Clinic: A Highly Efficacious, Off-Target-free, and Biomarker-Supported Strategy for fALS

Tommaso Iannitti, Joseph M. Scarrott, Shibi Likhite, +12

Molecular Therapy — Nucleic Acids

AAV9 Gene Therapy Increases Lifespan and Treats Pathological and Behavioral Abnormalities in a Mouse Model of CLN8-Batten Disease

Tyler B. Johnson, Katherine A. White, Jon J. Brudvig, +10

Molecular Therapy