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Effects of gene therapy on cardiovascular symptoms of lysosomal storage diseases

Edina Poletto1,2, Gabriela Pasqualim1,2, Roberto Giugliani1,2,3,4

  • 1Gene Therapy Center, Hospital de Clínicas de Porto Alegre, Porto Alegre, RS, Brazil.

Genetics and Molecular Biology
|May 28, 2019
PubMed

Insights

Gene therapy shows promise for treating heart disease caused by inherited lysosomal storage diseases (LSDs). This review explores various gene therapy approaches and their effects on cardiac function in animal models.

Area of Science:

  • Cardiovascular Medicine
  • Genetics
  • Biochemistry

Background:

  • Lysosomal storage diseases (LSDs) are inherited disorders impairing lysosomal function.
  • Substrate accumulation in LSDs leads to diverse clinical issues, notably cardiovascular disease and heart failure.
  • Current treatments are insufficient for addressing cardiac manifestations in LSDs.

Purpose of the Study:

  • To review gene therapy strategies for lysosomal storage diseases.
  • To evaluate the efficacy of gene therapy in ameliorating cardiac involvement in LSDs.
  • To focus on studies incorporating cardiac functional analysis in animal models.

Main Methods:

  • Review of existing literature on gene therapy for LSDs.
  • Analysis of studies employing animal models of LSDs.
  • Assessment of cardiac function parameters in treated models.

Main Results:

  • Gene therapy demonstrates potential in improving cardiac function in LSD models.
  • Different gene therapy approaches show varying degrees of success in addressing cardiac pathology.
  • Cardiac functional analysis is crucial for evaluating therapeutic outcomes.

Conclusions:

  • Gene therapy represents a promising therapeutic avenue for LSD-related heart disease.
  • Further research is needed to optimize gene therapy protocols for clinical application.
  • Targeted gene therapy may offer a viable solution for preventing and treating cardiac complications in LSD patients.

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