Cargando…

Promoterless, Nuclease‐Free Genome Editing Confers a Growth Advantage for Corrected Hepatocytes in Mice With Methylmalonic Acidemia

BACKGROUND AND AIMS: Adeno‐associated viral (AAV) gene therapy has shown great promise as an alternative treatment for metabolic disorders managed using liver transplantation, but remains limited by transgene loss and genotoxicity. Our study aims to test an AAV vector with a promoterless integrating...

Descripción completa

Detalles Bibliográficos
Autores principales:	Chandler, Randy J., Venturoni, Leah E., Liao, Jing, Hubbard, Brandon T., Schneller, Jessica L., Hoffmann, Victoria, Gordo, Susana, Zang, Shengwen, Ko, Chih‐Wei, Chau, Nelson, Chiang, Kyle, Kay, Mark A., Barzel, Adi, Venditti, Charles P.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	John Wiley and Sons Inc. 2021
Materias:	Original Articles
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8252383/ https://www.ncbi.nlm.nih.gov/pubmed/32976669 http://dx.doi.org/10.1002/hep.31570

Ejemplares similares

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

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

Promoterless gene targeting without nucleases ameliorates haemophilia B in mice
por: Barzel, A., et al.
Publicado: (2014)

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

Promoterless gene targeting without nucleases rescues lethality of a Crigler‐Najjar syndrome mouse model
por: Porro, Fabiola, et al.
Publicado: (2017)

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

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)

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

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

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

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)

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)

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

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

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

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)

Gene therapy in a murine model of Methylmalonic Acidemia (MMA) using rAAV9 mediated gene delivery
por: Sénac, J S, et al.
Publicado: (2011)

Neuropsychological endpoints for clinical trials in methylmalonic acidemia and propionic acidemia: A pilot study
por: Chapman, Kimberly A., et al.
Publicado: (2023)

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

Atypical imaging findings in the setting of methylmalonic acidemia in an infant
por: Chang, Warren, et al.
Publicado: (2015)

Clinical Course and Nutritional Management of Propionic and Methylmalonic Acidemias
por: Mobarak, Amira, et al.
Publicado: (2020)

Propionic and Methylmalonic Acidemias: Initial Clinical and Biochemical Presentation
por: Mobarak, Amira, et al.
Publicado: (2020)

Mitochondrial disease, mitophagy, and cellular distress in methylmalonic acidemia
por: Luciani, Alessandro, et al.
Publicado: (2021)

Multimodal MR imaging in acute exacerbation of methylmalonic acidemia
por: Saito, Riho, et al.
Publicado: (2023)

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

Methylmalonic acidemia mimicking diabetic ketoacidosis and septic shock in infants
por: Saini, Navdeep, et al.
Publicado: (2015)

Pulmonary embolism in a child with combined methylmalonic acidemia and homocystinuria
por: Chen, Lanqin, et al.
Publicado: (2018)

Long-term renal outcome in methylmalonic acidemia in adolescents and adults
por: Dao, Myriam, et al.
Publicado: (2021)

Efficacy of Living Donor Liver Transplantation in Patients with Methylmalonic Acidemia
por: Jang, Jae Guk, et al.
Publicado: (2021)

Methylmalonic Acidemia Masquerading as Rett Syndrome: An Atypical “Neurodegenerative” Variant?
por: Jayaram, Riddhee, 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)

Novel mutation in an Indian patient with Methylmalonic Acidemia, cblA type
por: Girisha, Katta Mohan, et al.
Publicado: (2012)

Affective Disorder as the First Manifestation of Methylmalonic Acidemia: A Case Report
por: Sheikhmoonesi, Fatemeh, et al.
Publicado: (2013)

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

Mutation analysis, treatment and prenatal diagnosis of Chinese cases of methylmalonic acidemia
por: Zhang, Chuan, et al.
Publicado: (2020)

Anesthetic management of a patient with methylmalonic acidemia: a case report
por: Uemura, Yuta, et al.
Publicado: (2018)

Segmental uniparental disomy of chromosome 4 in a patient with methylmalonic acidemia
por: Chen, Min, et al.
Publicado: (2019)

Ketamine and dexmedetomidine sedation for brain magnetic resonance imaging in methylmalonic acidemia
por: Srinivasa, Navyashree Krishnashastry, et al.
Publicado: (2021)

Cannot write session to /tmp/vufind_sessions/sess_vc4cs7vubv8ms7lk7fgbjrvpi5