Adeno-associated virus for cystic fibrosis gene therapy

Martini, Sabrina V.; Rocco, Patricia R. M.; Morales, Marcelo M.

doi:10.1590/s0100-879x2011007500123

Cited by 4 publications

(1 citation statement)

References 75 publications

(76 reference statements)

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“…AAV is unique in that it is naturally defective for replication and requires co-infection with a helper virus, most commonly adenovirus, to induce and support replication ( 119 ). This dependency on the helper virus for co-infection makes AAV non-pathogenic to humans ( 120 , 121 ). Its recombinant form does not encode any viral proteins and thus has low chances of being recognized by the host immune system.…”

Section: Ex Vivo Gene Therapymentioning

confidence: 99%

Gene therapy approaches for equine osteoarthritis

Thampi

Samulski²,

Grieger³

et al. 2022

Front. Vet. Sci.

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With an intrinsically low ability for self-repair, articular cartilage injuries often progress to cartilage loss and joint degeneration resulting in osteoarthritis (OA). Osteoarthritis and the associated articular cartilage changes can be debilitating, resulting in lameness and functional disability both in human and equine patients. While articular cartilage damage plays a central role in the pathogenesis of OA, the contribution of other joint tissues to the pathogenesis of OA has increasingly been recognized thus prompting a whole organ approach for therapeutic strategies. Gene therapy methods have generated significant interest in OA therapy in recent years. These utilize viral or non-viral vectors to deliver therapeutic molecules directly into the joint space with the goal of reprogramming the cells' machinery to secrete high levels of the target protein at the site of injection. Several viral vector-based approaches have demonstrated successful gene transfer with persistent therapeutic levels of transgene expression in the equine joint. As an experimental model, horses represent the pathology of human OA more accurately compared to other animal models. The anatomical and biomechanical similarities between equine and human joints also allow for the use of similar imaging and diagnostic methods as used in humans. In addition, horses experience naturally occurring OA and undergo similar therapies as human patients and, therefore, are a clinically relevant patient population. Thus, further studies utilizing this equine model would not only help advance the field of human OA therapy but also benefit the clinical equine patients with naturally occurring joint disease. In this review, we discuss the advancements in gene therapeutic approaches for the treatment of OA with the horse as a relevant patient population as well as an effective and commonly utilized species as a translational model.

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Section: Ex Vivo Gene Therapymentioning

confidence: 99%

Gene therapy approaches for equine osteoarthritis

Thampi

Samulski²,

Grieger³

et al. 2022

Front. Vet. Sci.

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Current World Literature

2012

Current Opinion in Pediatrics

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Perspectives on Genetic Medicine for Cystic Fibrosis

Smirnikhina

2022

CGT

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As any inherited protein deficiency disease, cystic fibrosis (CF) is a good candidate for gene replacement therapy. Despite the tremendous efforts of scientists around the world invested into development of this approach, it did not lead to the expected results for various reasons which are discussed in this review. At the same time, the emergence of new methods of genome editing, as well as their latest modifications, makes it possible to bypass some of the problems of “classical” CF gene therapy. The review surveys potential therapeutic agents for CF gene therapy, methods and routes of delivery, as well as discusses the problem of target cells for defect correction. Based on the results of these studies, editing genetic defects in the basal cells of the lungs and their counterparts in other organs will make it possible to create a drug for the treatment of CF with a single administration.

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Adeno-associated virus for cystic fibrosis gene therapy

Cited by 4 publications

References 75 publications

Gene therapy approaches for equine osteoarthritis

Gene therapy approaches for equine osteoarthritis

Current World Literature

Perspectives on Genetic Medicine for Cystic Fibrosis

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