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May 9, 2011Current Drug Discovery Technologies

Micro-RNA in Disease and Gene Therapy

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Authors

KLKenneth Lundström

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Overview

Review highlights microRNA dysregulation across diverse human diseases, suggesting therapeutic promise contingent on overcoming targeted viral and non-viral delivery hurdles.

Key Points

  • Summarize the regulatory roles of microRNAs across major cellular pathways and evaluate their involvement in human disease alongside delivery hurdles in gene therapy.
  • Synthesized molecular data on over 700 human microRNAs and their roles in transcription, cell cycle control, apoptosis, and neurogenesis.
  • Evaluated clinical associations from patient genetic analyses alongside delivery approaches including chemical modifications and viral vectors.
  • Absence or mutation of specific microRNAs associates broadly with cancer, neurodegenerative disorders, cardiovascular conditions, autoimmune diseases, and metabolic pathologies.
  • Clinical application in gene therapy remains restricted by difficulties in achieving sustained, targeted tissue delivery, prompting development of modified microRNAs and viral vector systems.

Cite This Study

Kenneth Lundström (2011) studied this question.

synapsesocial.com/papers/6a99b015d8169d122295b4fahttps://doi.org/10.2174/157016311795563857
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