Treatment of rare disorders by gene therapy



Gene therapy is a logical way to treat rare genetic disorders and cure a single gene defect by introducing with a 'correct' gene. The first gene-therapy trials were conducted using patients with rare monogenetic disorders, but these are now outstripped by the clinical testing of gene therapeutics for more common conditions, for ex: cancer, AIDS, and heart disease. This is partially due to a failure to achieve long-term gene expression with early vector systems, a critical condition for correcting many inborn genetic defects. 

  • Developing new probes for tissue targeting
  • Therapeutic bioengineering
  • Cellular mechano-biology
  • Tissue engineering for own stem cells
  • Bio engineering for medical diagnostic & imaging

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