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May 23, 2026Journal of Lipid and AtherosclerosisOpen Access

Molecular and Genetic Aspects of Lipid Metabolism Disorders and Potential Targets for Gene Therapy

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Key result

Gene and RNA therapies offer promising strategies for lipid disorders by targeting underlying genetic defects.

Why the study?

Understanding mechanisms of primary and secondary lipid metabolism disorders is essential to develop new therapeutic strategies, including gene therapy approaches.

Design

Review

Authors

EAElvira R. AkhmetzyanovaKazan Federal UniversityATANNA V. TIMOFEEVAKazan Federal UniversityDGDanil R. GallyamovKazan Federal University

Discussion

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Implication

Supports gene/RNA therapy development for lipid disorders; leaves open clinical adoption pending randomized trials.

Key Points

  • The aim is to explore the molecular and genetic factors involved in lipid metabolism disorders and identify potential gene therapy targets.
  • Review of current literature on lipid metabolism disorders and genetic mutations
  • Analysis of gene therapy advancements relevant to lipid metabolism
  • Summary of therapeutic strategies for both primary and secondary disorders.
  • Identified key genetic mutations related to lipid metabolism regulation.
  • Highlighted innovative gene therapy strategies that show promise for treating lipid disorders.
  • Summarized existing and potential therapeutic targets for future treatment options.

PICO

P
Population
Lipid metabolism disorders
I
Intervention / Comparator
Gene therapy and RNA-based therapeutics

This review summarizes the molecular and genetic mechanisms underlying lipid metabolism disorders and discusses potential targets for gene therapy.

Limitations

  • Concerns regarding safety and long-term efficacy
  • Ethical considerations
  • Potential immunogenicity of viral vectors and Cas9 protein
  • Limited packaging capacity of AAV vectors
  • Low-frequency genomic integration

Cite This Study

Akhmetzyanova et al. (2026) conducted a review in Lipid metabolism disorders. Gene therapy and RNA-based therapeutics was evaluated. Gene therapy and RNA-based therapeutics offer promising strategies for treating primary and secondary lipid metabolism disorders by targeting underlying genetic defects.

synapsesocial.com/papers/6a11439848a409a3a49de56ehttps://doi.org/10.12997/jla.2026.15.2.195

Topics

Molecular Mechanisms of Cardiac Remodeling
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