Antisense Oligonucleotides Delivered to the Mouse CNS Ameliorate Symptoms of Severe Spinal Muscular Atrophy

Marco A. Passini, Jie Bu, Amy M. Richards, +12

Science Translational Medicine

CNS-targeted gene therapy improves survival and motor function in a mouse model of spinal muscular atrophy

Marco A. Passini, Jie Bu, Eric M. Roskelley, +6

Journal of Clinical Investigation

Muscle FunctionImmunologyStandard Aav VectorsNeuroregenerationKinesiology +18

Augmenting CNS glucocerebrosidase activity as a therapeutic strategy for parkinsonism and other Gaucher-related synucleinopathies

S. Pablo Sardi, Jennifer Clarke, Catherine Viel, +11

Proceedings of the National Academy of Sciences

Intracranial Delivery of CLN2 Reduces Brain Pathology in a Mouse Model of Classical Late Infantile Neuronal Ceroid Lipofuscinosis

Marco A. Passini, James C. Dodge, Jie Bu, +13

Journal of Neuroscience

Transient expression of the NG2 proteoglycan by a subpopulation of activated macrophages in an excitotoxic hippocampal lesion

Jie Bu, Nadia Akhtar, Akiko Nishiyama

Glia

Elevated levels of the chemokine GRO-1 correlate with elevated oligodendrocyte progenitor proliferation in the jimpy mutant.

Qiulian Wu, R H Miller, Richard M. Ransohoff, +3

PubMed

Jp Spinal CordSpinal Cord InjuryDevelopmental BiologyElevated LevelsCell Regulation +13

Elevated Levels of the Chemokine GRO-1 Correlate with Elevated Oligodendrocyte Progenitor Proliferation in the<i>Jimpy</i>Mutant

Qian Wu, Robert H. Miller, Richard M. Ransohoff, +3

Journal of Neuroscience

Cell ProliferationSocial SciencesNeuroregenerationJp Spinal CordElevated Levels +25

Gene transfer of human acid sphingomyelinase corrects neuropathology and motor deficits in a mouse model of Niemann-Pick type A disease

James C. Dodge, Jennifer Clarke, Antonius Song, +11

Proceedings of the National Academy of Sciences

Neurological DisorderLysosomal Storage DiseaseImmunologyCellular NeurobiologySocial Sciences +17

AAV Vector-Mediated Correction of Brain Pathology in a Mouse Model of Niemann–Pick A Disease

Marco A. Passini, Shannon L. Macauley, Michael R. Huff, +9

Molecular Therapy

Translational Fidelity of Intrathecal Delivery of Self-Complementary AAV9–Survival Motor Neuron 1 for Spinal Muscular Atrophy

Marco A. Passini, Jie Bu, Amy M. Richards, +13

Human Gene Therapy

Knockout MouseNeuroregenerationSpinal Cord InjuryTranslational FidelityMedicine +11