Advanced Therapies for Inherited Diseases and Cancer: Recent Progress

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 ([email protected]) 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

Manuscript Submission Information

Manuscripts should be submitted online at www.mdpi.com by registering and logging in to this website. Once you are registered, click here to go to the submission form. Manuscripts can be submitted until the deadline. All submissions that pass pre-check are peer-reviewed. Accepted papers will be published continuously in the journal (as soon as accepted) and will be listed together on the special issue website. Research articles, review articles as well as short communications are invited. For planned papers, a title and short abstract (about 100 words) can be sent to the Editorial Office for announcement on this website.

Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). All manuscripts are thoroughly refereed through a single-blind peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. International Journal of Molecular Sciences is an international peer-reviewed open access semimonthly journal published by MDPI.

Please visit the Instructions for Authors page before submitting a manuscript. There is an Article Processing Charge (APC) for publication in this open access journal. For details about the APC please see here. Submitted papers should be well formatted and use good English. Authors may use MDPI's English editing service prior to publication or during author revisions.

• advanced therapies
• gene and cell therapy
• lentiviral and adeno-associated vectors
• CRISPR/Cas9
• iPSCs
• stem cells
• CAR-T
• personalized medicine
• inherited diseases
• rare diseases