Results
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761.
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Gene activation of SMN by selective disruption of lncRNA-mediated recruitment of PRC2 for the treatment of spinal muscular atrophy. [electronic resource] by
- Woo, Caroline J
- Maier, Verena K
- Davey, Roshni
- Brennan, James
- Li, Guangde
- Brothers, John
- Schwartz, Brian
- Gordo, Susana
- Kasper, Anne
- Okamoto, Trevor R
- Johansson, Hans E
- Mandefro, Berhan
- Sareen, Dhruv
- Bialek, Peter
- Chau, B Nelson
- Bhat, Balkrishen
- Bullough, David
- Barsoum, James
Producer: 20180412
In:
Proceedings of the National Academy of Sciences of the United States of America vol. 114
Availability: No items available.
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762.
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763.
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764.
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SMN2 copy number predicts acute or chronic spinal muscular atrophy but does not account for intrafamilial variability in siblings. [electronic resource] by
- Cuscó, I
- Barceló, M J
- Rojas-García, R
- Illa, I
- Gámez, J
- Cervera, C
- Pou, A
- Izquierdo, G
- Baiget, M
- Tizzano, E F
Producer: 20060929
In:
Journal of neurology vol. 253
Availability: No items available.
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765.
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In vitro restoration of functional SMN protein in human trophoblast cells affected by spinal muscular atrophy by small fragment homologous replacement. [electronic resource] by
- Sangiuolo, Federica
- Filareto, Antonio
- Spitalieri, Paola
- Scaldaferri, Maria Lucia
- Mango, Ruggiero
- Bruscia, Emanuela
- Citro, Gennaro
- Brunetti, Ercole
- De Felici, Massimo
- Novelli, Giuseppe
Producer: 20051215
In:
Human gene therapy vol. 16
Availability: No items available.
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766.
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767.
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768.
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769.
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Pharmacologically induced mouse model of adult spinal muscular atrophy to evaluate effectiveness of therapeutics after disease onset. [electronic resource] by
- Feng, Zhihua
- Ling, Karen K Y
- Zhao, Xin
- Zhou, Chunyi
- Karp, Gary
- Welch, Ellen M
- Naryshkin, Nikolai
- Ratni, Hasane
- Chen, Karen S
- Metzger, Friedrich
- Paushkin, Sergey
- Weetall, Marla
- Ko, Chien-Ping
Producer: 20161213
In:
Human molecular genetics vol. 25
Availability: No items available.
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770.
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771.
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772.
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773.
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774.
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775.
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776.
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777.
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778.
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Quantitative analysis of SMN1 and SMN2 genes based on DHPLC: a highly efficient and reliable carrier-screening test. [electronic resource] by
- Su, Yi-Ning
- Hung, Chia-Cheng
- Li, Hung
- Lee, Chien-Nan
- Cheng, Wen-Fang
- Tsao, Po-Nien
- Chang, Ming-Cheng
- Yu, Chia-Li
- Hsieh, Wu-Shiun
- Lin, Win-Li
- Hsu, Su-Ming
Producer: 20060605
In:
Human mutation vol. 25
Availability: No items available.
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779.
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780.
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