The First Viable Mouse Model of Combined Methylmalonic Acidemia and Homocystinuria, cblC Type Displays a Sustained Response to a Single Dose of AAV Gene Therapy and is Comparable to Chronic Hydroxocobalamin Administration

被引：0

作者：

Koutsoukos, Stefanos A. ^{[1
]}

Sloan, Jennifer L. ^{[1
]}

Murphy, Kelsey C. ^{[1
]}

Arnold, Madeline L. ^{[1
]}

Achilly, Nathan P. ^{[1
]}

Elliot, Gene ^{[2
]}

Zerfas, Patricia M. ^{[3
]}

Hoffmann, Victoria ^{[3
]}

Venditti, Charles P. ^{[1
]}

机构：

[1] NHGRI, Organ Acid Res Sect, Bethesda, MD 20892 USA

[2] NHGRI, Embryon Stem Cell & Transgen Mouse Core, Bethesda, MD 20892 USA

[3] NIH, Off Res Serv, Bldg 10, Bethesda, MD 20892 USA

来源：

MOLECULAR THERAPY | 2020年 / 28卷 / 04期

关键词：

D O I：

暂无

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

1348

引用

页码：578 / 578

页数：1

共 5 条

[1] Neonatal AAV Gene Therapy in a Viable Mouse Model of Combined Methylmalonic Acidemia and Homocystinuria, cblC Type, Is Comparable to Chronic Hydroxocobalamin Administration
Koutsoukos, Stefanos A.
Sloan, Jennifer L.
Murphy, Kelsey
Arnold, Madeline
Achilly, Nathan
Elliot, Gene
Zerfas, Patricia
Hoffmann, Victoria
Venditti, Charles P.
MOLECULAR THERAPY, 2021, 29 (04) : 252 - 253
[2] The First Viable Mouse Model of cblC Type Combined Methylmalonic Acidemia and Homocysteinemia: A Single Dose of AAV Rescues Neonatal Lethality and is Comparable to Chronic B12 Injection Therapy
Murphy, Kelsey C.
Sloan, Jennifer L.
Arnold, Madeline L.
Achilly, Nathan P.
Elliot, Gene
Zerfas, Patricia
Hoffman, Victoria
Venditti, Charles P.
MOLECULAR THERAPY, 2018, 26 (05) : 380 - 381
[3] The First Viable Mouse Model of cblC Type Combined Methylmalonic Acidemia and Hyperhomocysteinemia: AAV Gene Therapy Rescues Neonatal Lethality
Arnold, Madeline
Sloan, Jennifer
Achilly, Nathan
Elliot, Gene
Venditti, Charles
MOLECULAR THERAPY, 2017, 25 (05) : 113 - 113
[4] The First Viable Mouse Model of cblC Type Combined Methylmalonic Acidemia and Homocysteinemia: AAV Gene Therapy Rescues Neonatal Lethality and Provides Insight into Disease-Associated Retinal Degeneration
Arnold, Madeline
Sloan, Jennifer L.
Achilly, Nathan P.
Elliot, Gene
Onojafe, Ighovie F.
Brooks, Brian P.
Venditti, Charles P.
MOLECULAR THERAPY, 2016, 24 : S20 - S21
[5] Systemic AAV Gene Therapy for cblC Type Combined Methylmalonic Acidemia and Homocysteinemia Rescues Neonatal Lethality and Provides Lasting Phenotypic and Metabolic Correction Comparable to Chronic OHCbl Injections
Murphy, Kelsey C.
Sloan, Jennifer L.
Arnold, Madeline L.
Achilly, Nathan P.
Elliot, Gene
Zerfas, Patricia
Hoffman, Victoria
Venditti, Charles P.
MOLECULAR THERAPY, 2019, 27 (04) : 203 - 204

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