Cargando…

Gene therapy in a murine model of Methylmalonic Acidemia (MMA) using rAAV9 mediated gene delivery

Methylmalonic acidemia (MMA), an inherited metabolic disorder caused by deficient activity of methylmalonyl-CoA mutase (MUT), carries a poor prognosis for long-term survival. While administration of a recombinant adeno-associated virus serotype 8 vector (rAAV8) can rescue Mut(-/-) mice from neonatal...

Descripción completa

Detalles Bibliográficos
Autores principales:	Sénac, J S, Chandler, R J, Sysol, J R, Li, L, Venditti, C P
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	2011
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3382069/ https://www.ncbi.nlm.nih.gov/pubmed/21776024 http://dx.doi.org/10.1038/gt.2011.108

Ejemplares similares

Renal Growth in Isolated Methylmalonic Acidemia (MMA)
por: Kruszka, Paul S., et al.
Publicado: (2013)

A critical reappraisal of dietary practices in methylmalonic acidemia raises concerns about the safety of medical foods. Part 1: Isolated methylmalonic acidemias (MMA)
por: Manoli, Irini, et al.
Publicado: (2015)

Elevated methylmalonic acidemia (MMA) screening markers in Hispanic and preterm newborns
por: Peng, Gang, et al.
Publicado: (2018)

Intravenous rAAV2/9 injection for murine cochlear gene delivery
por: Shibata, Seiji B., et al.
Publicado: (2017)

Uncommonly Missed Diagnosis of Methylmalonic Acidemia (MMA) in Adults and Usefulness of Testing for MMA in Cases of Seizures/Neuropathy/Weakness/Ataxia
por: Cocuzzo, Brianna, et al.
Publicado: (2023)

Long-term Rescue of a Lethal Murine Model of Methylmalonic Acidemia Using Adeno associated Viral Gene Therapy
por: Chandler, Randy J, et al.
Publicado: (2009)

Management of methylmalonic acidemia (MMA) with N‐carbamylglutamate: A case report from Italy
por: Tubili, Flavia, et al.
Publicado: (2022)

Long-term effectiveness of carglumic acid in patients with propionic acidemia (PA) and methylmalonic acidemia (MMA): a randomized clinical trial
por: Alfadhel, Majid, et al.
Publicado: (2021)

Central nervous system-targeted adeno-associated virus gene therapy in methylmalonic acidemia
por: May, Francis J., et al.
Publicado: (2021)

Systemic gene therapy for methylmalonic acidemia using the novel adeno-associated viral vector 44.9
por: Chandler, Randy J., et al.
Publicado: (2022)

Systematic literature review and meta-analysis on the epidemiology of methylmalonic acidemia (MMA) with a focus on MMA caused by methylmalonyl-CoA mutase (mut) deficiency
por: Almási, Tímea, et al.
Publicado: (2019)

1-(13)C-propionate breath testing as a surrogate endpoint to assess efficacy of liver-directed therapies in methylmalonic acidemia (MMA)
por: Manoli, Irini, et al.
Publicado: (2021)

Methylmalonic Acidemia with Novel MUT Gene Mutations
por: Panigrahi, Inusha, et al.
Publicado: (2017)

In vivo genome editing at the albumin locus to treat methylmalonic acidemia
por: Schneller, Jessica L., et al.
Publicado: (2021)

Correction to: 1-(13)C-propionate breath testing as a surrogate endpoint to assess efficacy of liver-directed therapies in methylmalonic acidemia (MMA)
por: Manoli, Irini, et al.
Publicado: (2021)

Evaluation of long-term effectiveness of the use of carglumic acid in patients with propionic acidemia (PA) or methylmalonic acidemia (MMA): study protocol for a randomized controlled trial
por: Nashabat, Marwan, et al.
Publicado: (2019)

Metabolic phenotype of methylmalonic acidemia in mice and humans: the role of skeletal muscle
por: Chandler, Randy J, et al.
Publicado: (2007)

Methylmalonic acidemia: Neurodevelopment and neuroimaging
por: Chen, Tao, et al.
Publicado: (2023)

ImmTOR nanoparticles enhance AAV transgene expression after initial and repeat dosing in a mouse model of methylmalonic acidemia
por: Ilyinskii, Petr O., et al.
Publicado: (2021)

Robust Long-term Transduction of Common Marmoset Neuromuscular Tissue With rAAV1 and rAAV9
por: Okada, Hironori, et al.
Publicado: (2013)

Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia
por: An, Ding, et al.
Publicado: (2017)

Association of Methylmalonic Acidemia and Erythema Nodosum
por: Karamizadeh, Zohre, et al.
Publicado: (2011)

The Value of Liver Transplantation for Methylmalonic Acidemia
por: Jiang, Yi-Zhou, et al.
Publicado: (2019)

Extracorporeal Delivery of rAAV with Metabolic Exchange and Oxygenation
por: Bieber, Scott, et al.
Publicado: (2013)

Systemic IGF-1 gene delivery by rAAV9 improves spontaneous autoimmune peripheral polyneuropathy (SAPP)
por: Gao, Tong, et al.
Publicado: (2018)

Gene Delivery to Adipose Tissue Using Transcriptionally Targeted rAAV8 Vectors
por: Uhrig-Schmidt, Silke, et al.
Publicado: (2014)

rAAV8 and rAAV9-Mediated Long-Term Muscle Transduction with Tacrolimus (FK506) in Non-Human Primates
por: Ishii, Akiko, et al.
Publicado: (2020)

Durability of transgene expression after rAAV gene therapy
por: Muhuri, Manish, et al.
Publicado: (2022)

Methylmalonic acidemia and diabetic ketoacidosis: An unusual association
por: Kumar, Suresh
Publicado: (2015)

Corrigendum: The Value of Liver Transplantation for Methylmalonic Acidemia
por: Jiang, Yi-Zhou, et al.
Publicado: (2020)

Biomarkers for drug development in propionic and methylmalonic acidemias
por: Longo, Nicola, et al.
Publicado: (2022)

rAAV9 combined with renal vein injection is optimal for kidney-targeted gene delivery: conclusion of a comparative study
por: Rocca, Céline J., et al.
Publicado: (2014)

Threshold for Pre-existing Antibody Levels Limiting Transduction Efficiency of Systemic rAAV9 Gene Delivery: Relevance for Translation
por: Meadows, Aaron S., et al.
Publicado: (2019)

Noninvasive prenatal diagnosis in a fetus at risk for methylmalonic acidemia
por: Gu, Wei, et al.
Publicado: (2014)

Molecular Signature of Astrocytes for Gene Delivery by the Synthetic Adeno‐Associated Viral Vector rAAV9P1
por: Bauer, Amelie, et al.
Publicado: (2022)

Systemic gene therapy using an AAV44.9 vector rescues a neonatal lethal mouse model of propionic acidemia
por: Chandler, Randy J., et al.
Publicado: (2023)

Novel AAV-mediated genome editing therapy improves health and survival in a mouse model of methylmalonic acidemia
por: Zhang, Shengwen, et al.
Publicado: (2022)

PEG-mediated transduction of rAAV as a platform for spatially confined and efficient gene delivery
por: Zou, Liang, et al.
Publicado: (2022)

Long term physiologic modification using rAAV in utero gene-therapy
por: Garrett, Deiadra J, et al.
Publicado: (2004)

Proposed guidelines for the diagnosis and management of methylmalonic and propionic acidemia
por: Baumgartner, Matthias R, et al.
Publicado: (2014)

Cannot write session to /tmp/vufind_sessions/sess_vfhv2koutah7p29o7rufq3g05g