Gene Therapy for Duchenne Muscular Dystrophy

Rodino‐Klapac, Louise R.; Chicoine, Louis G.; Kaspar, Brian K.; Mendell, Jerry R.

doi:10.1001/archneur.64.9.1236

Cited by 50 publications

(26 citation statements)

References 47 publications

(26 reference statements)

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“…128 The mutant protein responsible is dystrophin and several different mutations or deletions in its large gene have been documented to give rise to a variety of different degrees of clinical manifestations. 129 In severe forms, the protein is significantly truncated or even absent. Importantly, it has been shown that introduction of a dystrophin minigene can substantially alleviate the clinical symptoms.…”

Section: Gene Targeting Monogenetic Causes Of Hfmentioning

confidence: 99%

Gene Therapy in Heart Failure

Vinge

Raake

Koch

2008

Circulation Research

120

111

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Abstract-With increasing knowledge of basic molecular mechanisms governing the development of heart failure (HF), the possibility of specifically targeting key pathological players is evolving. Technology allowing for efficient in vivo transduction of myocardial tissue with long-term expression of a transgene enables translation of basic mechanistic knowledge into potential gene therapy approaches. Gene therapy in HF is in its infancy clinically with the predominant amount of experience being from animal models. Nevertheless, this challenging and promising field is gaining momentum as recent preclinical studies in larger animals have been carried out and, importantly, there are 2 newly initiated phase I clinical trials for HF gene therapy. To put it simply, 2 parameters are needed for achieving success with HF gene therapy: (1) clearly identified detrimental/beneficial molecular targets; and (2) the means to manipulate these targets at a molecular level in a sufficient number of cardiac cells. However, several obstacles do exist on our way to efficient and safe gene transfer to human myocardium. Some of these obstacles are discussed in this review; however, it primarily focuses on the molecular target systems that have been subjected to intense investigation over the last decade in an attempt to make gene therapy for human HF a reality.

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Section: Gene Targeting Monogenetic Causes Of Hfmentioning

confidence: 99%

Gene Therapy in Heart Failure

Vinge

Raake

Koch

2008

Circulation Research

120

111

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“…Thus, Galgt2 overexpression can inhibit muscular dystrophy in two very different models of muscular dystrophy, a finding that suggests its mechanism of action may be more broadly applicable than other approaches such as gene replacement. Here we show that overexpression of Galgt2 mediated by a recombinant adenoassociated vector confers protection against loss of muscle force during eccentric contractions, not only in mdx muscles but also in WT muscles, and that it is as effective as gene replacement with microdystrophin (17), a shortened version of the dystrophin protein that is able to be packaged into AAV gene therapy vectors that are currently preferred for translational gene therapy research (41,42). Thus, Galgt2 overexpression may have therapeutic impact in many types of muscle injury.…”

mentioning

confidence: 92%

Overexpression ofGalgt2in skeletal muscle prevents injury resulting from eccentric contractions in both mdx and wild-type mice

Martin¹,

Xu²,

Rodino‐Klapac³

et al. 2009

American Journal of Physiology-Cell Physiology

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107

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“…The remaining erythrocytes have a molecular weight greater than Ficol and deposited in test tubes. [11][12][13][14][15][16][17][18] The supernatant, which contained the mono nuclear cells, was removed, and the 400 Gera was centrifuged for 12 minutes. Finally, the sediment cell, the antibody and Neuroglial cells was added after 34 minutes incubation at 5 °C, the cell mixture was passed from pillar LSMACS.…”

Section: Methodsmentioning

confidence: 99%