Gene Therapy for Rare Diseases
A special issue of Cells (ISSN 2073-4409). This special issue belongs to the section "Cell Nuclei: Function, Transport and Receptors".
Deadline for manuscript submissions: 30 April 2025 | Viewed by 7782
Special Issue Editor
Interests: Rho GTPases; keratinocytes; mouse disease models
Special Issues, Collections and Topics in MDPI journals
Special Issue Information
Dear Colleagues,
Each rare disease is infrequent, but since more than 7000 rare diseases exist, more than 300 million patients are suffering from rare diseases worldwide, making them an important unmet health problem. Most rare diseases are caused by mutations. Therefore, gene therapy is the only curative treatment possibility for patients with rare diseases. Stimulated by the development of CRISPR gene editing and improved viral vector technologies, many trials are currently ongoing to correct the defective genes of patients with rare diseases and improve their quality of life. On the other hand, many challenges still need to be overcome to make gene therapy a standard treatment option in the clinic. These challenges include the efficient targeting of relevant stem cells, the effectiveness of precise genome editing, and patient safety. The recent development of prime editing and base editing has further contributed to the excitement in the field and to the hope that a cure for many rare diseases is possible in the near future. This Special Issue will accept reviews and original research articles in the field of gene editing and rare diseases.
Prof. Dr. Cord Brakebusch
Guest Editor
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Keywords
- gene therapy
- gene editing
- rare diseases
- CRISPR
- viral vectors
- base editing
- prime editing
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