Gene Therapy for Rare Diseases
Gene therapy is a logical way to treat rare inheritable conditions; cure a single gene disfigurement by introducing a' correct' gene. The first gene- remedy trials were conducted using cases with rare monogenetic diseases, but these are now outnumbered by the clinical testing of gene therapeutics for more common conditions, such as cancer, AIDS and cardiovascular disease. due to a failure to achieve long- term gene expression with early vector systems, a critical demand for correcting numerous inborn inheritable blights. Now, with the arrival of adeno- associated viral( AAV) and lentiviral vectors, which demonstrate persistent gene expression in animal studies, this technological hedge may have been overcome.
Related Conference of Gene Therapy for Rare Diseases
September 22-23, 2025
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Gene Therapy for Rare Diseases Conference Speakers
Recommended Sessions
- Different Types of Rare Diseases
- Gene Therapy for Rare Diseases
- Orphan Drugs, Their Potencies and Current Researches
- Pediatric Infectious Diseases
- Rare Aging and Endocrine Disease
- Rare Diseases and Immune System
- Rare Diseases and Their Causes
- Rare Diseases in Cancer and Treatment
- Rare Genetic Disorders
- Rare Mental and Behavioral Disorders
- Rare Neurological Disorders
- Rare Oral Diseases
- Rare Skin Diseases
- Sexually transmitted diseases
- Viral Infectious Diseases
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