Gene Therapy in Monogenic Congenital Myopathies

Guan, Xuan; Goddard, Melissa; Mack, David L.; Childers, Martin K.

doi:10.1016/b978-0-12-800548-4.00013-9

Cited by 2 publications

(1 citation statement)

References 102 publications

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“…A cell uses its membrane to communicate with the outside world; thus, most membrane protein‐targeting therapeutics work to alter cellular signalling. In the case of membrane protein defects, replacement of the faulty proteins by gene therapy or protein replacement may be the only solution [131]. However, there are potential risks and limitations associated with gene therapy, and the clinical application of protein therapy has been hampered by the lack of effective vehicles and issues with protein production [132–134].…”

Section: Therapeutic Applications Of Surface‐modified Evsmentioning

confidence: 99%

Extracellular vesicles as a platform for membrane‐associated therapeutic protein delivery

Yang

Hong

Cho

et al. 2018

J of Extracellular Vesicle

181

148

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Membrane proteins are of great research interest, particularly because they are rich in targets for therapeutic application. The suitability of various membrane proteins as targets for therapeutic formulations, such as drugs or antibodies, has been studied in preclinical and clinical studies. For therapeutic application, however, a protein must be expressed and purified in as close to its native conformation as possible. This has proven difficult for membrane proteins, as their native conformation requires the association with an appropriate cellular membrane. One solution to this problem is to use extracellular vesicles as a display platform. Exosomes and microvesicles are membranous extracellular vesicles that are released from most cells. Their membranes may provide a favourable microenvironment for membrane proteins to take on their proper conformation, activity, and membrane distribution; moreover, membrane proteins can cluster into microdomains on the surface of extracellular vesicles following their biogenesis. In this review, we survey the state-of-the-art of extracellular vesicle (exosome and small-sized microvesicle)-based therapeutics, evaluate the current biological understanding of these formulations, and forecast the technical advances that will be needed to continue driving the development of membrane protein therapeutics.

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Section: Therapeutic Applications Of Surface‐modified Evsmentioning

confidence: 99%

Extracellular vesicles as a platform for membrane‐associated therapeutic protein delivery

Yang

Hong

Cho

et al. 2018

J of Extracellular Vesicle

181

148

View full text Add to dashboard Cite

show abstract

Limb-girdle Muscular Dystrophy and Therapy: Insights into Cell and Gene-based Approaches

Taheri

Taghizadeh

Pour

et al. 2020

CGT

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The Limb-Girdle Muscular Dystrophies (LGMD) are genetically heterogeneous disorders, responsible for muscle wasting and severe form of dystrophies. Despite the critical developments in the insight and information of pathomechanisms of limb-girdle muscular dystrophy, any definitive treatments do not exist, and current strategies are only based on the improvement of the signs of disorder and to enhance the life quality without resolving an underlying cause. There is a crucial relationship between pharmacological therapy and different consequences; therefore, other treatment strategies will be required. New approaches, such as gene replacement, gene transfer, exon skipping, siRNA knockdown, and anti-myostatin therapy, which can target specific cellular or molecular mechanism of LGMD, could be a promising avenue for the treatment. Recently, genome engineering strategies with a focus on molecular tools such as CRISPR-Cas9 are used to different types of neuromuscular disorders and show the highest potential for clinical translation of these therapies. Thus, recent advancements and challenges in the field will be reviewed in this paper.

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Gene Therapy in Monogenic Congenital Myopathies

Cited by 2 publications

References 102 publications

Extracellular vesicles as a platform for membrane‐associated therapeutic protein delivery

Extracellular vesicles as a platform for membrane‐associated therapeutic protein delivery

Limb-girdle Muscular Dystrophy and Therapy: Insights into Cell and Gene-based Approaches

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