Gene Therapy

It is a technique which aims at treating diseases by genetically modifying population of cells into the patient body instead of using drugs and surgery to prevent from any disorder. Various methods have been developed for the gene delivery to the target cells which includes viral and non-viral systems. It is a creation of producing new cells that will produce a specific neurotransmitter which is dopamine to protect the neural system and then these cells are transplanted to the patient having Parkinsons disease because the neurons cannot be renewed further. Some important reasons are there for focusing gene therapy which includes firstly there is no cure for Parkinson’s disease; secondly some genes which are identified can modulate the neuron phenotype.

Gene therapy clinical trials in PD have focused on:

  • Restoring dopamine synthesis
  • Neuroprotection
  • Genetic neuromodulation
  • Addressing disease-specific pathogenic variants

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