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Effects of gene therapy on cardiovascular symptoms of lysosomal storage diseases.

ABSTRACT: Lysosomal storage diseases (LSDs) are inherited conditions caused by impaired lysosomal function and consequent substrate storage, leading to a range of clinical manifestations, including cardiovascular disease. This may lead to significant symptoms and even cardiac failure, which is an important cause of death among patients. Currently available treatments do not completely correct cardiac involvement in the LSDs. Gene therapy has been tested as a therapeutic alternative with promising results for the heart disease. In this review, we present the results of different approaches of gene therapy for LSDs, mainly in animal models, and its effects in the heart, focusing on protocols with cardiac functional analysis.

SUBMITTER: Poletto E 

PROVIDER: S-EPMC6687348 | biostudies-literature | 2019

REPOSITORIES: biostudies-literature

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Effects of gene therapy on cardiovascular symptoms of lysosomal storage diseases.

Poletto Edina E   Pasqualim Gabriela G   Giugliani Roberto R   Matte Ursula U   Baldo Guilherme G  

Genetics and molecular biology 20190523 1 suppl 1

Lysosomal storage diseases (LSDs) are inherited conditions caused by impaired lysosomal function and consequent substrate storage, leading to a range of clinical manifestations, including cardiovascular disease. This may lead to significant symptoms and even cardiac failure, which is an important cause of death among patients. Currently available treatments do not completely correct cardiac involvement in the LSDs. Gene therapy has been tested as a therapeutic alternative with promising results  ...[more]

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