Rare Diseases and Orphan Drugs

Gene Therapy for Rare Diseases

Gene therapy may be a logical thanks to treat rare genetic diseases; cure one gene defect by introducing a 'correct' gene. The main quality treatment preliminaries were led utilizing patients with uncommon monogenetic messes, yet these are presently dwarfed by the clinical testing of quality therapeutics for more normal conditions, for example, malignancy, AIDS and cardiovascular sickness. This is partly thanks to a failure to realize long-term organic phenomenon with early vector systems, a critical requirement for correcting many inborn genetic defects. Now, with the arrival of adeno-associated viral (AAV) and lentiviral vectors, which demonstrate persistent organic phenomenon in animal studies, this technological barrier may are overcome. These vectors are anticipated to make the idea of the many gene-therapy protocols for inherited genetic diseases.

Related Associations of Rare Diseases Research:

Alzheimer's disease Organizations, Ann & Robert H. Lurie Children’s Hospital, Birmingham children’s Hospital, Boston Children’s Hospital, Canadian Organization for Rare Diseases, Chicago Rare Disease Foundation, Children’s hospital of Pittsburgh, National Alliances for Rare Diseases, National Institute of Health (NIH), National Organization for Rare Diseases, NDC Medicine, Rare Diseases Patient Association Funding, Rare Diseases South Africa, Rare Diseases Translational Research Collaboration.