Cargando…

Amelioration of Muscle and Nerve Pathology in LAMA2 Muscular Dystrophy by AAV9-Mini-Agrin

LAMA2-related muscular dystrophy (LAMA2 MD) is the most common and fatal form of early-onset congenital muscular dystrophies. Due to the large size of the laminin α2 cDNA and heterotrimeric structure of the protein, it is challenging to develop a gene-replacement therapy. Our group has developed a n...

Descripción completa

Detalles Bibliográficos
Autores principales:	Qiao, Chunping, Dai, Yi, Nikolova, Viktoriya D., Jin, Quan, Li, Jianbin, Xiao, Bin, Li, Juan, Moy, Sheryl S., Xiao, Xiao
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2018
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5948311/ https://www.ncbi.nlm.nih.gov/pubmed/29766020 http://dx.doi.org/10.1016/j.omtm.2018.01.005

Ejemplares similares

Apoptosis inhibitors and mini-agrin have additive benefits in congenital muscular dystrophy mice
por: Meinen, Sarina, et al.
Publicado: (2011)

Amelioration of muscle and nerve pathology of Lama2-related dystrophy by AAV9-laminin-αLN linker protein
por: McKee, Karen K., et al.
Publicado: (2022)

Evaluation of an AAV9-mini-dystrophin gene therapy candidate in a rat model of Duchenne muscular dystrophy
por: Le Guiner, Caroline, et al.
Publicado: (2023)

LAMA2 Nonsense Variant in an Italian Greyhound with Congenital Muscular Dystrophy
por: Christen, Matthias, et al.
Publicado: (2021)

CRISPRa-induced upregulation of human LAMA1 compensates for LAMA2-deficiency in Merosin-deficient congenital muscular dystrophy
por: Arockiaraj, Annie I., et al.
Publicado: (2023)

Cobblestone Malformation in LAMA2 Congenital Muscular Dystrophy (MDC1A)
por: Jayakody, Himali, et al.
Publicado: (2020)

Congenital muscular dystrophy in a dog with a LAMA2 gene deletion
por: Shelton, G. Diane, et al.
Publicado: (2021)

Nerve pathology is prevented by linker proteins in mouse models for LAMA2-related muscular dystrophy
por: Reinhard, Judith R, et al.
Publicado: (2023)

Novel LAMA2 Gene Mutations Associated with Merosin-Deficient Congenital Muscular Dystrophy
por: Hashemi-Gorji, Feyzollah, et al.
Publicado: (2018)

LAMA2‐related muscular dystrophy: Natural history of a large pediatric cohort
por: Zambon, Alberto A., et al.
Publicado: (2020)

Zebrafish Models of LAMA2-Related Congenital Muscular Dystrophy (MDC1A)
por: Fabian, Lacramioara, et al.
Publicado: (2020)

Fibrogenesis in LAMA2-Related Muscular Dystrophy Is a Central Tenet of Disease Etiology
por: Accorsi, Anthony, et al.
Publicado: (2020)

Laminin and Integrin in LAMA2-Related Congenital Muscular Dystrophy: From Disease to Therapeutics
por: Barraza-Flores, Pamela, et al.
Publicado: (2020)

Identification of Two Novel LAMA2 Mutations in a Chinese Patient with Congenital Muscular Dystrophy
por: Zhou, Jing, et al.
Publicado: (2018)

Novel Homozygous Pathogenic Mutations of LAMA 2 Gene in Patients with Congen ital Muscular Dystrophy
por: KHODAENIA, Negar, et al.
Publicado: (2021)

Widening the spectrum of LAMA 2 congenital muscular dystrophy (MDC1A): cobblestone malformation
por: Borella, Luiz Fernando Monte, et al.
Publicado: (2021)

Limb girdle muscular dystrophy 23 caused by compound heterozygous mutations of LAMA2 gene
por: Xu, Yuqing, et al.
Publicado: (2023)

LAMA2-Related Muscular Dystrophy Across the Life Span: A Cross-sectional Study
por: Bouman, Karlijn, et al.
Publicado: (2023)

Natural history and genetic study of LAMA2-related muscular dystrophy in a large Chinese cohort
por: Tan, Dandan, et al.
Publicado: (2021)

Absence of microRNA-21 does not reduce muscular dystrophy in mouse models of LAMA2-CMD
por: Moreira Soares Oliveira, Bernardo, et al.
Publicado: (2017)

A Mosaic Mutation in the LAMA2 Gene in a Case of Merosin-deficient Congenital Muscular Dystrophy
por: Chausova, P. A., et al.
Publicado: (2021)

Vemurafenib improves muscle histopathology in a mouse model of LAMA2-related congenital muscular dystrophy
por: Oliveira-Santos, Ariany, et al.
Publicado: (2023)

Liver-specific microRNA-122 target sequences incorporated in AAV vectors efficiently inhibits transgene expression in the liver
por: Qiao, Chunping, et al.
Publicado: (2010)

Systemic AAV Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy
por: Duan, Dongsheng
Publicado: (2018)

Novel compound heterozygous mutations of LAMA2-limb–girdle muscular dystrophy: A case report and literature review
por: Wang, Duo-Zi, et al.
Publicado: (2023)

Novel Mutation (c.8725T>C) in Two Siblings With Late-Onset LAMA2-Related Muscular Dystrophy
por: Kim, Min-Wook, et al.
Publicado: (2017)

Merosin-deficient congenital muscular dystrophy type 1a: detection of LAMA2 variants in Vietnamese patients
por: Tran, Van Khanh, et al.
Publicado: (2023)

Unique genotype-phenotype correlations within LAMA2-related limb girdle muscular dystrophy in Chinese patients
por: Huang, Xiuli, et al.
Publicado: (2023)

Long-term Evaluation of AAV-CRISPR Genome Editing for Duchenne Muscular Dystrophy
por: Nelson, Christopher E., et al.
Publicado: (2019)

Assessment of Therapeutic Potential of a Dual AAV Approach for Duchenne Muscular Dystrophy
por: Albini, Sonia, et al.
Publicado: (2023)

Rare variant in LAMA2 gene causing congenital muscular dystrophy in a Sudanese family. A case report
por: AMIN, MUTAZ, et al.
Publicado: (2019)

Brain Dysfunction in LAMA2-Related Congenital Muscular Dystrophy: Lessons From Human Case Reports and Mouse Models
por: Arreguin, Andrea J., et al.
Publicado: (2020)

Evidence of Two Novel LAMA2 Variants in a Patient With Muscular Dystrophy: Facing the Challenges of a Certain Diagnosis
por: Meyer, Stefanie, et al.
Publicado: (2022)

Case report: Novel frameshift mutation in LAMA2 gene causing congenital muscular dystrophy type 1A
por: Diaz-Lombana, Natalia, et al.
Publicado: (2023)

Strategies for Bottlenecks of rAAV-Mediated Expression in Skeletal and Cardiac Muscle of Duchenne Muscular Dystrophy
por: Li, Na, et al.
Publicado: (2022)

Outcome Measures for COL6 and LAMA2-Related Dystrophies
por: Schwaede, Abigail N., et al.
Publicado: (2020)

Deletion of exon 4 in LAMA2 is the most frequent mutation in Chinese patients with laminin α2-related muscular dystrophy
por: Ge, Lin, et al.
Publicado: (2018)

Case report: Adult-onset limb girdle muscular dystrophy in sibling pair due to novel homozygous LAMA2 missense variant
por: Katz, Matthew, et al.
Publicado: (2023)

Immunity and AAV-Mediated Gene Therapy for Muscular Dystrophies in Large Animal Models and Human Trials
por: Wang, Zejing, et al.
Publicado: (2011)

Disease Rescue and Increased Lifespan in a Model of Cardiomyopathy and Muscular Dystrophy by Combined AAV Treatments
por: Vitiello, Carmen, et al.
Publicado: (2009)

Cannot write session to /tmp/vufind_sessions/sess_lk141as9om4rr6tkthq5mjf11r