[PDF][PDF] Sustained therapeutic reversal of Huntington's disease by transient repression of huntingtin synthesis
…, JW Artates, A Weiss, SH Cheng, LS Shihabuddin… - Neuron, 2012 - cell.com
The primary cause of Huntington's disease (HD) is expression of huntingtin with a
polyglutamine expansion. Despite an absence of consensus on the mechanism(s) of toxicity, …
polyglutamine expansion. Despite an absence of consensus on the mechanism(s) of toxicity, …
Adult spinal cord stem cells generate neurons after transplantation in the adult dentate gyrus
LS Shihabuddin, PJ Horner, J Ray… - Journal of …, 2000 - Soc Neuroscience
The adult rat spinal cord contains cells that can proliferate and differentiate into astrocytes
and oligodendroglia in situ. Using clonal and subclonal analyses we demonstrate that, in …
and oligodendroglia in situ. Using clonal and subclonal analyses we demonstrate that, in …
Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy
…, G Hung, EM Kaye, LS Shihabuddin… - Science translational …, 2011 - science.org
Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disorder caused
by mutations in the SMN1 gene that result in a deficiency of SMN protein. One approach to …
by mutations in the SMN1 gene that result in a deficiency of SMN protein. One approach to …
[HTML][HTML] iPSC-derived neurons from GBA1-associated Parkinson's disease patients show autophagic defects and impaired calcium homeostasis
…, U Hedrich, D Berg, LS Shihabuddin… - Nature …, 2014 - nature.com
Mutations in the acid β-glucocerebrosidase (GBA1) gene, responsible for the lysosomal
storage disorder Gaucher’s disease (GD), are the strongest genetic risk factor for Parkinson’s …
storage disorder Gaucher’s disease (GD), are the strongest genetic risk factor for Parkinson’s …
Acid β‐glucosidase mutants linked to Gaucher disease, Parkinson disease, and Lewy body dementia alter α‐synuclein processing
…, SH Cheng, GA Grabowski, LS Shihabuddin… - Annals of …, 2011 - Wiley Online Library
… Here, we explored the still unknown mechanism(s) that underlie(s) the gene's association
… access by its intracellular protease(s), referred to as synucleinase(s). Intriguingly, a recently …
… access by its intracellular protease(s), referred to as synucleinase(s). Intriguingly, a recently …
CNS expression of glucocerebrosidase corrects α-synuclein pathology and memory in a mouse model of Gaucher-related synucleinopathy
…, SH Cheng, LS Shihabuddin - Proceedings of the …, 2011 - National Acad Sciences
Emerging genetic and clinical evidence suggests a link between Gaucher disease and the
synucleinopathies Parkinson disease and dementia with Lewy bodies. Here, we provide …
synucleinopathies Parkinson disease and dementia with Lewy bodies. Here, we provide …
[HTML][HTML] CNS-targeted gene therapy improves survival and motor function in a mouse model of spinal muscular atrophy
…, KW Klinger, LS Shihabuddin… - The Journal of …, 2010 - Am Soc Clin Investig
Spinal muscular atrophy (SMA) is a neuromuscular disease caused by a deficiency of
survival motor neuron (SMN) due to mutations in the SMN1 gene. In this study, an adeno-…
survival motor neuron (SMN) due to mutations in the SMN1 gene. In this study, an adeno-…
Augmenting CNS glucocerebrosidase activity as a therapeutic strategy for parkinsonism and other Gaucher-related synucleinopathies
…, SH Cheng, LS Shihabuddin - Proceedings of the …, 2013 - National Acad Sciences
Mutations of GBA1, the gene encoding glucocerebrosidase, represent a common genetic
risk factor for developing the synucleinopathies Parkinson disease (PD) and dementia with …
risk factor for developing the synucleinopathies Parkinson disease (PD) and dementia with …
FGF-2 is sufficient to isolate progenitors found in the adult mammalian spinal cord
LS Shihabuddin, J Ray, FH Gage - Experimental neurology, 1997 - Elsevier
The adult rat brain contains progenitor cells that can be induced to proliferatein vitroin
response to FGF-2. In the present study we explored whether similar progenitor cells can be …
response to FGF-2. In the present study we explored whether similar progenitor cells can be …
Gain of toxic function by long-term AAV9-mediated SMN overexpression in the sensorimotor circuit
…, Y Recinos, E Workman, LS Shihabuddin… - Nature …, 2021 - nature.com
The neurodegenerative disease spinal muscular atrophy (SMA) is caused by deficiency in
the survival motor neuron (SMN) protein. Currently approved SMA treatments aim to restore …
the survival motor neuron (SMN) protein. Currently approved SMA treatments aim to restore …