Rare Diseases: From Molecular Pathways to Therapeutic Strategies 2.0
A special issue of Biomolecules (ISSN 2218-273X). This special issue belongs to the section "Molecular Medicine".
Deadline for manuscript submissions: 30 September 2024 | Viewed by 508
Special Issue Editor
Interests: lysosomal storage disease; fabry disease; autophagy; gene therapy; inflammation; genetic disorders; adeno associated virus
Special Issues, Collections and Topics in MDPI journals
Special Issue Information
Dear Colleagues,
In recent decades, a massive improvement in biotechnological tools has paved the way to more efficient gene therapy strategies, which are currently under clinical trials or have successfully reached commercial authorisation. Gene therapy is a promising approach to cure many inborn errors of metabolism and neuromuscular disorders, as well as other rare disorders without a present effective treatment.
The scientific community is more and more focused on the preclinical development of new possible gene therapy strategies, and the number of clinical trials for gene-therapy-based drugs has increased considerably with the aim of repeating the successful results of the recently approved drugs for Spinal Muscular Atrophy, Leber’s Congenital Amaurosis, and Metachromatic leukodystrophy.
The scope of this Special Issue is to describe the latest advances concerning new gene-therapy-based treatments, mainly focusing on metabolic diseases of genetic origin and neuromuscular disorders.
This Special Issue will welcome original research articles and up-to-date reviews on the described topics.
We look forward to reading your contributions.
Dr. Saida Ortolano
Guest Editor
Manuscript Submission Information
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Keywords
- adeno-associated virus
- gene editing
- metabolic diseases
- lysosomal disorders
- CRISPR
- inborn errors of metabolism
- neuromuscular disease
