Short-term treatment of golden retriever muscular dystrophy (GRMD) dogs with rAAVrh74.MHCK7.GALGT2 induces muscle glycosylation and utrophin expression but has no significant effect on muscle strength.

Martin, Paul T; Zygmunt, Deborah A; Ashbrook, Anna; Hamilton, Sonia; Packer, Davin; Birch, Sharla M; Bettis, Amanda K; Balog-Alvarez, Cynthia J; Guo, Lee-Jae; Nghiem, Peter P; Kornegay, Joe N

Martin, Paul T; Zygmunt, Deborah A; Ashbrook, Anna; Hamilton, Sonia; Packer, Davin; Birch, Sharla M; Bettis, Amanda K; Balog-Alvarez, Cynthia J; Guo, Lee-Jae; Nghiem, Peter P; Kornegay, Joe N.

Afiliação

Martin PT; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, Ohio, United States of America.
Zygmunt DA; Department of Pediatrics, The Ohio State University College of Medicine, Columbus, Ohio, United States of America.
Ashbrook A; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, Ohio, United States of America.
Hamilton S; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, Ohio, United States of America.
Packer D; Neuroscience Undergraduate Program, The Ohio State University, Columbus, Ohio, United States of America.
Birch SM; Neuroscience Graduate Program, The Ohio State University, Columbus, Ohio, United States of America.
Bettis AK; Department of Veterinary Integrative Biosciences, College of Veterinary Medicine and Biomedical Sciences, Texas A&M University, College Station, TX, United States of America.
Balog-Alvarez CJ; Department of Veterinary Integrative Biosciences, College of Veterinary Medicine and Biomedical Sciences, Texas A&M University, College Station, TX, United States of America.
Guo LJ; Department of Veterinary Integrative Biosciences, College of Veterinary Medicine and Biomedical Sciences, Texas A&M University, College Station, TX, United States of America.
Nghiem PP; Department of Veterinary Integrative Biosciences, College of Veterinary Medicine and Biomedical Sciences, Texas A&M University, College Station, TX, United States of America.
Kornegay JN; Department of Veterinary Integrative Biosciences, College of Veterinary Medicine and Biomedical Sciences, Texas A&M University, College Station, TX, United States of America.

PLoS One ; 16(3): e0248721, 2021.

Article em En | MEDLINE | ID: mdl-33770101

Assuntos

Doenças do Cão/terapia; Distrofina/genética; Terapia Genética; Glicosiltransferases/farmacologia; Distrofias Musculares/terapia; Distrofia Muscular de Duchenne/terapia; Animais; Modelos Animais de Doenças; Doenças do Cão/genética; Doenças do Cão/patologia; Cães; Distroglicanas/biossíntese; Distroglicanas/genética; Distrofina/biossíntese; Expressão Gênica/efeitos dos fármacos; Glicosilação/efeitos dos fármacos; Glicosiltransferases/genética; Humanos; Força Muscular/efeitos dos fármacos; Músculo Esquelético/efeitos dos fármacos; Músculo Esquelético/patologia; Distrofias Musculares/genética; Distrofias Musculares/patologia; Distrofia Muscular de Duchenne/genética; Distrofia Muscular de Duchenne/patologia; Utrofina/genética

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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Terapia Genética / Distrofina / Glicosiltransferases / Distrofia Muscular de Duchenne / Doenças do Cão / Distrofias Musculares Limite: Animals / Humans Idioma: En Revista: PLoS One Assunto da revista: CIENCIA / MEDICINA Ano de publicação: 2021 Tipo de documento: Article País de afiliação: Estados Unidos

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