Functional Genomics and Gene Therapy for Rare Diseases
Functional genomics aims to understand the roles of genes and their interactions, providing critical insights for rare disease treatment. Gene therapy offers the potential to correct genetic abnormalities, with viral vector-based delivery and CRISPR technology paving the way for innovative solutions. Clinical trials focus on ensuring the safety, efficacy, and durability of these treatments. As research progresses, refining delivery methods and addressing ethical considerations remain essential for wider adoption.
Related Conference of Functional Genomics and Gene Therapy for Rare Diseases
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Functional Genomics and Gene Therapy for Rare Diseases Conference Speakers
Recommended Sessions
- Advancement in the Diagnosis of Rare Diseases
- Biomarkers for Rare Disease Diagnosis and Treatment
- Biomedical Research
- Challenges of Living with a Rare Disease
- Clinical Case Reports and Case Studies
- Clinical Research on Orphan Drugs
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- Drug Discovery for Rare Diseases
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- Genetic Testing and Counselling of Rare Diseases
- Health Economics and Market Access for Orphan Drugs
- Infectious Diseases and Immune Deficiencies
- Introduction and Causes of New Rare Diseases
- Orphan Drugs Potentiality
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- Rare Neurological Disorders
- Rare Nutritional and Metabolic Diseases
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- Rare Skin Diseases
- Regulatory Considerations for Developing Orphan Drugs
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