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Rationally designed AAV2 and AAVrh8R capsids provide improved transduction in the retina and brain. [electronic resource] by
- Sullivan, Jennifer A
- Stanek, Lisa M
- Lukason, Michael J
- Bu, Jie
- Osmond, Shayla R
- Barry, Elizabeth A
- O'Riordan, Catherine R
- Shihabuddin, Lamya S
- Cheng, Seng H
- Scaria, Abraham
Producer: 20181105
In:
Gene therapy vol. 25
Availability: No items available.
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8.
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Marked differences in neurochemistry and aggregates despite similar behavioural and neuropathological features of Huntington disease in the full-length BACHD and YAC128 mice. [electronic resource] by
- Pouladi, Mahmoud A
- Stanek, Lisa M
- Xie, Yuanyun
- Franciosi, Sonia
- Southwell, Amber L
- Deng, Yu
- Butland, Stefanie
- Zhang, Weining
- Cheng, Seng H
- Shihabuddin, Lamya S
- Hayden, Michael R
Producer: 20120913
In:
Human molecular genetics vol. 21
Availability: No items available.
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9.
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Widespread AAV1- and AAV2-mediated transgene expression in the nonhuman primate brain: implications for Huntington's disease. [electronic resource] by
- Hadaczek, Piotr
- Stanek, Lisa
- Ciesielska, Agnieszka
- Sudhakar, Vivek
- Samaranch, Lluis
- Pivirotto, Philip
- Bringas, John
- O'Riordan, Catherine
- Mastis, Bryan
- San Sebastian, Waldy
- Forsayeth, John
- Cheng, Seng H
- Bankiewicz, Krystof S
- Shihabuddin, Lamya S
Producer: 20160713
In:
Molecular therapy. Methods & clinical development vol. 3
Availability: No items available.
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10.
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CNS expression of glucocerebrosidase corrects alpha-synuclein pathology and memory in a mouse model of Gaucher-related synucleinopathy. [electronic resource] by
- Sardi, S Pablo
- Clarke, Jennifer
- Kinnecom, Cathrine
- Tamsett, Thomas J
- Li, Lingyun
- Stanek, Lisa M
- Passini, Marco A
- Grabowski, Gregory A
- Schlossmacher, Michael G
- Sidman, Richard L
- Cheng, Seng H
- Shihabuddin, Lamya S
Producer: 20111003
In:
Proceedings of the National Academy of Sciences of the United States of America vol. 108
Availability: No items available.
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11.
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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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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
Availability: No items available.
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13.
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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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14.
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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
Availability: No items available.
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