Gene therapy for rare diseases
Gene Therapy mainly involves the introduction or alteration of genetic material within a cell or organism with an intention of curing the disease. Both gene therapy and cell therapy are overlapping fields of biomedical research with the goals of repairing the direct cause of Genetic diseases in DNA or cellular population respectively, the discovery of recombinant DNA technology in the 1970s provided tools to develop gene therapy efficiently. Scientists use these techniques to readily manipulate viral genomes, isolate genes and identify mutations involved in human diseases, characterize and regulate gene expressions, and engineer various non- viral and viral vectors. Various long-term treatments for anaemia, haemophilia, cystic fibrosis, muscular dystrophy, Gauscher’s disease, lysosomal storage diseases, cardiovascular diseases, diabetes and diseases of bones and joints are resolved through successful gene therapy.
- Gene therapy for malignant melanoma
- Development of regenerative treatment models
- Gene therapy for sickle-cell disease
- Different vectors for gene therapy
- Gene therapy products
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Gene therapy for rare diseases Conference Speakers
Recommended Sessions
- Allergic and immunologic disorders
- Challenges in rare disease development
- Clinical trials and treatment
- Gene therapy for rare diseases
- Infectious diseases
- Orphan Diseases and public health
- Orphan drugs potentiality
- Orphan medicinal products
- Rare diseases in blood stream
- Rare infectious diseases and immune deficiencies
- Rare mental and behavioral diseases
- Rare neurological diseases
- Rare nutritional and metabolic diseases
- Rare pediatric diseases
- Treatment and advanced therapies for rare diseases
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