Surrogate gene therapy for muscular dystrophy

An engineered truncated gene derived from the dystrophin-related protein (utrophin), prevents pathology without an immune response in an animal model of Duchenne muscular dystrophy (DMD) gene therapy.

Bibliográfalaš dieđut
Váldodahkkit:	Davies, KE, Chamberlain, JS
Materiálatiipa:	Journal article
Giella:	English
Almmustuhtton:	Springer Nature 2019

Geahča maid

Therapeutic approaches to muscular dystrophy.
Dahkki: Goyenvalle, A, et al.
Almmustuhtton: (2011)

The Pathogenesis and Therapy of Muscular Dystrophies
Dahkki: Guiraud, S, et al.
Almmustuhtton: (2015)

Progress in therapy for Duchenne muscular dystrophy.
Dahkki: Fairclough, R, et al.
Almmustuhtton: (2011)

Progress in therapy for Duchenne muscular dystrophy
Dahkki: Fairclough, R, et al.
Almmustuhtton: (2011)

Utrophin in the therapy of Duchenne Muscular Dystrophy
Dahkki: Potter, A, et al.
Almmustuhtton: (2006)

From diagnosis to therapy in Duchenne muscular dystrophy
Dahkki: Babbs, A, et al.
Almmustuhtton: (2020)

The allure of stem cell therapy for muscular dystrophy.
Dahkki: Grounds, MD, et al.
Almmustuhtton: (2007)

Immunity and AAV-mediated gene therapy for muscular dystrophies in large animal models and human trials
Dahkki: Zejing eWang, et al.
Almmustuhtton: (2011-09-01)

Micro-dystrophin genes bring hope of an effective therapy for duchenne muscular dystrophy
Dahkki: Davies, K, et al.
Almmustuhtton: (2019)

Therapeutic approaches for Duchenne muscular dystrophy
Dahkki: Roberts, TC, et al.
Almmustuhtton: (2023)

Muscular dystrophy: from gene to patient.
Dahkki: Hopkins, J, et al.
Almmustuhtton: (2000)

The role of utrophin in the potential therapy of Duchenne muscular dystrophy.
Dahkki: Perkins, K, et al.
Almmustuhtton: (2002)

Prepare for the era of gene therapy for Duchenne muscular dystrophy
Dahkki: Cheng ZHANG
Almmustuhtton: (2019-05-01)

Advance and cogitation of gene therapy for Duchenne muscular dystrophy
Dahkki: Cheng ZHANG, et al.
Almmustuhtton: (2019-05-01)

PABPN1 gene therapy for oculopharyngeal muscular dystrophy
Dahkki: A. Malerba, et al.
Almmustuhtton: (2017-03-01)

Pharmacological strategies for muscular dystrophy.
Dahkki: Khurana, T, et al.
Almmustuhtton: (2003)

Multi-parametric MRI at 14T for muscular dystrophy mice treated with AAV vector-mediated gene therapy.
Dahkki: Joshua Park, et al.
Almmustuhtton: (2015-01-01)

Muscular dystrophy--reason for optimism?
Dahkki: Burton, E, et al.
Almmustuhtton: (2002)

DUCHENNE MUSCULAR DYSTROPHY FROM GENE DIAGNOSIS TO MOLECULAR THERAPY
Dahkki: , Masafumi Matsuo
Almmustuhtton: (2003)

Duchenne muscular dystrophy from gene diagnosis to molecular therapy
Dahkki: , Masafumi Matsuo
Almmustuhtton: (2003)

Cardiac therapies for Duchenne muscular dystrophy
Dahkki: Md Nur Ahad Shah, et al.
Almmustuhtton: (2023-07-01)

Rehabilitation therapy of Duchenne muscular dystrophy
Dahkki: Cheng ZHANG, et al.
Almmustuhtton: (2012-06-01)

Regenerative biomarkers for Duchenne muscular dystrophy
Dahkki: Guiraud, S, et al.
Almmustuhtton: (2019)

Severe cardiac and skeletal manifestations in DMD-edited microminipigs: an advanced surrogate for Duchenne muscular dystrophy
Dahkki: Masayoshi Otake, et al.
Almmustuhtton: (2024-05-01)

Treating muscular dystrophy with stem cells?
Dahkki: Davies, K, et al.
Almmustuhtton: (2006)

Regenerative biomarkers for Duchenne muscular dystrophy
Dahkki: Simon Guiraud, et al.
Almmustuhtton: (2019-01-01)

Muscular Dystrophy /
Dahkki: Naff, Clay Farris, editor 192865, et al.
Almmustuhtton: (2012)

The potential of utrophin and dystrophin combination therapies for Duchenne muscular dystrophy
Dahkki: Guiraud, S, et al.
Almmustuhtton: (2019)

Therapy for Duchenne muscular dystrophy: renewed optimism from genetic approaches.
Dahkki: Fairclough, R, et al.
Almmustuhtton: (2013)

Utrophin upregulation in Duchenne muscular dystrophy.
Dahkki: Hirst, R, et al.
Almmustuhtton: (2005)

Duchenne muscular dystrophy - past, present and future
Dahkki: Davies, K
Almmustuhtton: (2002)

Pharmacological advances for treatment in Duchenne muscular dystrophy
Dahkki: Guiraud, S, et al.
Almmustuhtton: (2017)

Regional localization of the murine Duchenne muscular dystrophy gene on the mouse X chromosome.
Dahkki: Chamberlain, J, et al.
Almmustuhtton: (1987)

Natural history of Duchenne muscular dystrophy
Dahkki: Qing KE, et al.
Almmustuhtton: (2015-05-01)

Gene Editing to Tackle Facioscapulohumeral Muscular Dystrophy
Dahkki: Virginie Mariot, et al.
Almmustuhtton: (2022-07-01)

Zebrafish orthologs of human muscular dystrophy genes
Dahkki: Zon Leonard I, et al.
Almmustuhtton: (2007-03-01)

Muscular dystrophy associated with the DMD gene in women
Dahkki: E. O. Vorontsova, et al.
Almmustuhtton: (2024-09-01)

Identification of new chemical compounds with upregulate utrophin for the therapy of Duchenne muscular dystrophy
Dahkki: Fairclough, R, et al.
Almmustuhtton: (2012)

Micro-dystrophin gene therapy prevents heart failure in an improved Duchenne muscular dystrophy cardiomyopathy mouse model
Dahkki: Zachary M. Howard, et al.
Almmustuhtton: (2021-04-01)