Advanced Therapies for Inherited Diseases and Cancer: Recent Progress
A special issue of International Journal of Molecular Sciences (ISSN 1422-0067). This special issue belongs to the section "Molecular Pathology, Diagnostics, and Therapeutics".
Deadline for manuscript submissions: closed (20 February 2025) | Viewed by 1953
Special Issue Editors
Interests: rare diseases; congenital coagulopathies; factor V deficiency; gene and cell therapy; CRISPR/Cas9; lentiviral vectors
Interests: hemostasis; rare bleeding disorders; factor XI deficiency; antithrombin deficiency; thrombosis; congenital disorders of glycosylation; anticoagulant therapy; gene therapy
Special Issues, Collections and Topics in MDPI journals
Special Issue Information
Dear Colleagues,
Contemporary pharmacology is nowadays taking a new direction with the application of the new (or advanced) therapies for the treatment of inherited diseases and cancer. The new targets INCLUDE the mutational causes and the antigen components of cancer cell membranes. Gene therapy, through CRISPR/Cas9 gene editing and viral vectors, and cell therapy, through induced pluripotent stem cells (iPSCs), stem cells and CAR-T therapy, are the new strategies set to revolutionize the way patients are treated. Classical pharmacology will gradually make way for new therapeutic protocols based on personalized and individualized medicine, from recent breakthroughs in the fields of proteomics and genomics. Advanced therapies will bring a fresh ray of hope to many hereditary conditions (including rare or ultra-rare diseases) and many types of cancer. Severe combined immunodeficiency, congenital coagulopathies such as hemophilia A and B, deficiency of factor V or other coagulation factors, cystic fibrosis, Duchenne muscular dystrophy, familial hypercholesterolemia, diabetes, several kinds of hemoglobinopathies, Fanconi anemia, neurogenerative diseases, malignant melanoma, blindness, some types of leukemia such as acute B-cell lymphoblastic leukemia or chronic lymphocytic leukemia, and refractory lymphomas are just a few of the many conditions that will benefit from the new therapies.
Dr. Juan Andres de Pablo Moreno (jdepablo@ucm.es) from Complutense University of Madrid is serving as the co-Guest Editor and will assist Dr. Antonio Liras and Dr. Maria Eugenia de la Morena-Barrio in managing this Special Issue.
Prof. Dr. Antonio Liras
Dr. Maria Eugenia De la Morena-Barrio
Guest Editors
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Keywords
- advanced therapies
- gene and cell therapy
- lentiviral and adeno-associated vectors
- CRISPR/Cas9
- iPSCs
- stem cells
- CAR-T
- personalized medicine
- inherited diseases
- rare diseases
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