A PHASE 1/2 CLINICAL TRIAL FOR AAV8-MEDIATED LIVER-DIRECTED GENE THERAPY IN ADULTS WITH LATE-ONSET OTC DEFICIENCY: INTERIM RESULTS FROM COHORT 1

被引：0

作者：

Harding, Cary O. ^{[1
]}

Geberhiwot, Tarekegn ^{[2
]}

Aldamiz-Echevarria, Luis ^{[3
]}

Luz Couce, Maria ^{[4
]}

Khan, Anal ^{[5
]}

Tan, Wen-Hann ^{[6
]}

Crombez, Eric ^{[7
]}

Diaz, George A. ^{[8
]}

机构：

[1] Oregon Hlth & Sci Univ, Portland, OR 97201 USA

[2] Univ Birmingham, Birmingham, W Midlands, England

[3] Hosp Univ Cruces, Baracaldo, Spain

[4] Univ Santiago de Compostela, Santiago De Compostela, Spain

[5] Univ Calgary, Calgary, AB, Canada

[6] Harvard Med Sch, Boston Childrens Hosp, Boston, MA 02115 USA

[7] Ultragenyx Gene Therapy, Cambridge, MA USA

[8] Icahn Sch Med Mt Sinai, New York, NY 10029 USA

来源：

MOLECULAR GENETICS AND METABOLISM | 2019年 / 126卷 / 03期

关键词：

D O I：

暂无

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

引用

页码：278 / 278

页数：1

共 50 条

[1] A PHASE 1/2 CLINICAL TRIAL FOR AAV8-MEDIATED LIVER-DIRECTED GENE THERAPY IN ADULTS WITH LATE-ONSET OTC DEFICIENCY: INTERIM RESULTS FROM COHORT 1
Harding, Cary O.
Geberhiwot, Tarekegn
Aldamiz-Echevarria, Luis
Luz Couce, Maria
Khan, Aneal
Tan, Wen-Hann
Crombez, Eric
Diaz, George A.
[J]. MOLECULAR GENETICS AND METABOLISM, 2019, 127 (03) : 240 - 240
[2] A phase 1/2 clinical trial for AAV8-mediated liver-directed gene therapy in adults with late-onset OTC deficiency
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Aldamiz-Echevarria, L.
Couce, M. L.
Harding, C. O.
Khan, A.
Tan, W. H.
Lin, J.
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Diaz, G. A.
[J]. HUMAN GENE THERAPY, 2018, 29 (12) : A59 - A59
[3] Preliminary Results from Cohorts 1 and 2 of CAPtivate: A Phase 1/2 Clinical Trial of AAV8-Mediated Liver-Directed Gene Therapy in Adults with Late-Onset OTC Deficiency
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[J]. MOLECULAR THERAPY, 2019, 27 (04) : 2 - 3
[4] DESIGN OF AN OPEN LABEL PHASE 1/2 CLINICAL TRIAL FOR AAV8-MEDIATED LIVER-DIRECTED GENE THERAPY IN ADULTS WITH LATE ONSET ORNITHINE TRANSCARBAMYLASE DEFICIENCY
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Lin, Julie
Crombez, Eric
[J]. MOLECULAR GENETICS AND METABOLISM, 2018, 123 (03) : 198 - 198
[5] A phase 1/2 clinical trial of AAV8 gene therapy in adults with late-onset OTC deficiency: CAPtivate cohort 1+2 results
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Lee, C.
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[J]. HUMAN GENE THERAPY, 2019, 30 (11) : A18 - A18
[6] AAV8 Gene Therapy as a Potential Treatment in Adults with Late-Onset OTC Deficiency: Results from a Phase 1/2 Clinical Trial
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[J]. MOLECULAR THERAPY, 2020, 28 (04) : 222 - 222
[7] Positive Cohort 1 Results from the Phase 1/2 Trial with AAV8-Mediated Liver-Directed Gene Therapy in Adults with Glycogen Storage Disease Type Ia
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[J]. MOLECULAR THERAPY, 2019, 27 (04) : 12 - 12
[8] COHORT 1-4 RESULTS FROM A PHASE 1/2 CLINICAL TRIAL OF AN AAV8-MEDIATED LIVER-DIRECTED GENE THERAPY IN ADULTS WITH GLYCOGEN STORAGE DISEASE TYPE IA (GSDIA)
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[J]. MOLECULAR GENETICS AND METABOLISM, 2022, 135 (04) : 296 - 296
[9] Positive cohort 1 results from the phase 1/2, AAV8-mediated liver-directed gene therapy trial in glycogen storage disease type Ia (GSDIa)
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[J]. HUMAN GENE THERAPY, 2019, 30 (11) : A14 - A14
[10] AAV8 Gene Therapy as a Potential Treatment in Adults with Late-Onset Ornithine Transcarbamylase (OTC) Deficiency: Updated Results from a Phase 1/2 Clinical Trial
Harding, Cary O.
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[J]. MOLECULAR THERAPY, 2021, 29 (04) : 17 - 17

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