Results
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41.
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Sustained therapeutic reversal of Huntington's disease by transient repression of huntingtin synthesis. [electronic resource] by
- Kordasiewicz, Holly B
- Stanek, Lisa M
- Wancewicz, Edward V
- Mazur, Curt
- McAlonis, Melissa M
- Pytel, Kimberly A
- Artates, Jonathan W
- Weiss, Andreas
- Cheng, Seng H
- Shihabuddin, Lamya S
- Hung, Gene
- Bennett, C Frank
- Cleveland, Don W
Producer: 20120914
In:
Neuron vol. 74
Availability: No items available.
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42.
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Gene transfer of human acid sphingomyelinase corrects neuropathology and motor deficits in a mouse model of Niemann-Pick type A disease. [electronic resource] by
- Dodge, James C
- Clarke, Jennifer
- Song, Antonius
- Bu, Jie
- Yang, Wendy
- Taksir, Tatyana V
- Griffiths, Denise
- Zhao, Michael A
- Schuchman, Edward H
- Cheng, Seng H
- O'Riordan, Catherine R
- Shihabuddin, Lamya S
- Passini, Marco A
- Stewart, Gregory R
Producer: 20060118
In:
Proceedings of the National Academy of Sciences of the United States of America vol. 102
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43.
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Augmenting CNS glucocerebrosidase activity as a therapeutic strategy for parkinsonism and other Gaucher-related synucleinopathies. [electronic resource] by
- Sardi, S Pablo
- Clarke, Jennifer
- Viel, Catherine
- Chan, Monyrath
- Tamsett, Thomas J
- Treleaven, Christopher M
- Bu, Jie
- Sweet, Lindsay
- Passini, Marco A
- Dodge, James C
- Yu, W Haung
- Sidman, Richard L
- Cheng, Seng H
- Shihabuddin, Lamya S
Producer: 20130502
In:
Proceedings of the National Academy of Sciences of the United States of America vol. 110
Availability: No items available.
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44.
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Extensive Transduction and Enhanced Spread of a Modified AAV2 Capsid in the Non-human Primate CNS. [electronic resource] by
- Naidoo, Jerusha
- Stanek, Lisa M
- Ohno, Kousaku
- Trewman, Savanah
- Samaranch, Lluis
- Hadaczek, Piotr
- O'Riordan, Catherine
- Sullivan, Jennifer
- San Sebastian, Waldy
- Bringas, John R
- Snieckus, Christopher
- Mahmoodi, Amin
- Mahmoodi, Amir
- Forsayeth, John
- Bankiewicz, Krystof S
- Shihabuddin, Lamya S
Producer: 20190814
In:
Molecular therapy : the journal of the American Society of Gene Therapy vol. 26
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45.
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Timing of therapeutic intervention determines functional and survival outcomes in a mouse model of late infantile batten disease. [electronic resource] by
- Cabrera-Salazar, Mario A
- Roskelley, Eric M
- Bu, Jie
- Hodges, Bradley L
- Yew, Nelson
- Dodge, James C
- Shihabuddin, Lamya S
- Sohar, Istvan
- Sleat, David E
- Scheule, Ronald K
- Davidson, Beverly L
- Cheng, Seng H
- Lobel, Peter
- Passini, Marco A
Producer: 20080107
In:
Molecular therapy : the journal of the American Society of Gene Therapy vol. 15
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46.
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Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy. [electronic resource] by
- Passini, Marco A
- Bu, Jie
- Richards, Amy M
- Kinnecom, Cathrine
- Sardi, S Pablo
- Stanek, Lisa M
- Hua, Yimin
- Rigo, Frank
- Matson, John
- Hung, Gene
- Kaye, Edward M
- Shihabuddin, Lamya S
- Krainer, Adrian R
- Bennett, C Frank
- Cheng, Seng H
Producer: 20110613
In:
Science translational medicine vol. 3
Availability: No items available.
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47.
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Acid β-glucosidase mutants linked to Gaucher disease, Parkinson disease, and Lewy body dementia alter α-synuclein processing. [electronic resource] by
- Cullen, Valerie
- Sardi, S Pablo
- Ng, Juliana
- Xu, You-Hai
- Sun, Ying
- Tomlinson, Julianna J
- Kolodziej, Piotr
- Kahn, Ilana
- Saftig, Paul
- Woulfe, John
- Rochet, Jean-Christophe
- Glicksman, Marcie A
- Cheng, Seng H
- Grabowski, Gregory A
- Shihabuddin, Lamya S
- Schlossmacher, Michael G
Producer: 20110822
In:
Annals of neurology vol. 69
Availability: No items available.
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48.
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Partial rescue of some features of Huntington Disease in the genetic absence of caspase-6 in YAC128 mice. [electronic resource] by
- Wong, Bibiana K Y
- Ehrnhoefer, Dagmar E
- Graham, Rona K
- Martin, Dale D O
- Ladha, Safia
- Uribe, Valeria
- Stanek, Lisa M
- Franciosi, Sonia
- Qiu, Xiaofan
- Deng, Yu
- Kovalik, Vlad
- Zhang, Weining
- Pouladi, Mahmoud A
- Shihabuddin, Lamya S
- Hayden, Michael R
Producer: 20151214
In:
Neurobiology of disease vol. 76
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49.
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Combination brain and systemic injections of AAV provide maximal functional and survival benefits in the Niemann-Pick mouse. [electronic resource] by
- Passini, Marco A
- Bu, Jie
- Fidler, Jonathan A
- Ziegler, Robin J
- Foley, Joseph W
- Dodge, James C
- Yang, Wendy W
- Clarke, Jennifer
- Taksir, Tatyana V
- Griffiths, Denise A
- Zhao, Michael A
- O'Riordan, Catherine R
- Schuchman, Edward H
- Shihabuddin, Lamya S
- Cheng, Seng H
Producer: 20070730
In:
Proceedings of the National Academy of Sciences of the United States of America vol. 104
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50.
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Intracranial delivery of CLN2 reduces brain pathology in a mouse model of classical late infantile neuronal ceroid lipofuscinosis. [electronic resource] by
- Passini, Marco A
- Dodge, James C
- Bu, Jie
- Yang, Wendy
- Zhao, Qi
- Sondhi, Dolan
- Hackett, Neil R
- Kaminsky, Stephen M
- Mao, Qinwen
- Shihabuddin, Lamya S
- Cheng, Seng H
- Sleat, David E
- Stewart, Gregory R
- Davidson, Beverly L
- Lobel, Peter
- Crystal, Ronald G
Producer: 20060427
In:
The Journal of neuroscience : the official journal of the Society for Neuroscience vol. 26
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51.
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Delivery of AAV-IGF-1 to the CNS extends survival in ALS mice through modification of aberrant glial cell activity. [electronic resource] by
- Dodge, James C
- Haidet, Amanda M
- Yang, Wendy
- Passini, Marco A
- Hester, Mark
- Clarke, Jennifer
- Roskelley, Eric M
- Treleaven, Christopher M
- Rizo, Liza
- Martin, Heather
- Kim, Soo H
- Kaspar, Rita
- Taksir, Tatyana V
- Griffiths, Denise A
- Cheng, Seng H
- Shihabuddin, Lamya S
- Kaspar, Brian K
Producer: 20080819
In:
Molecular therapy : the journal of the American Society of Gene Therapy vol. 16
Availability: No items available.
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52.
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iPSC-derived neurons from GBA1-associated Parkinson's disease patients show autophagic defects and impaired calcium homeostasis. [electronic resource] by
- Schöndorf, David C
- Aureli, Massimo
- McAllister, Fiona E
- Hindley, Christopher J
- Mayer, Florian
- Schmid, Benjamin
- Sardi, S Pablo
- Valsecchi, Manuela
- Hoffmann, Susanna
- Schwarz, Lukas Kristoffer
- Hedrich, Ulrike
- Berg, Daniela
- Shihabuddin, Lamya S
- Hu, Jing
- Pruszak, Jan
- Gygi, Steven P
- Sonnino, Sandro
- Gasser, Thomas
- Deleidi, Michela
Producer: 20151203
In:
Nature communications vol. 5
Availability: No items available.
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53.
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Translational fidelity of intrathecal delivery of self-complementary AAV9-survival motor neuron 1 for spinal muscular atrophy. [electronic resource] by
- Passini, Marco A
- Bu, Jie
- Richards, Amy M
- Treleaven, Christopher M
- Sullivan, Jennifer A
- O'Riordan, Catherine R
- Scaria, Abraham
- Kells, Adrian P
- Samaranch, Lluis
- San Sebastian, Waldy
- Federici, Thais
- Fiandaca, Massimo S
- Boulis, Nicholas M
- Bankiewicz, Krystof S
- Shihabuddin, Lamya S
- Cheng, Seng H
Producer: 20150313
In:
Human gene therapy vol. 25
Availability: No items available.
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