Thomas Weber, PhD Email Thomas Weber
- ASSOCIATE PROFESSOR | Medicine, Cardiology
- ASSOCIATE PROFESSOR | Microbiology
- ASSOCIATE PROFESSOR | Cell, Developmental & Regenerative Biology
Cardiovascular, Cell Biology, Gene Therapy, Intracellular Transport, Protein Trafficking & Sorting, Regeneration, Viruses and Virology
Multi-Disciplinary Training Area
BS, College Schaffhausen
MSc, Swiss Federal Institute of Technology
Dr sc nat (PhD), Swiss Federal Institute of Technology
Postdoctorate, Memorial Sloan Kettering Cancer Center
Adeno-Associated Virus (AAV) Biology and AAV Gene Therapy
Gene therapy is a promising platform to treat and potentially cure currently intractable disorders. Among the most promising gene delivery vehicles are vectors based on adeno-associated virus (AAV).
AAV is particularly attractive because:
1) It is not known to be associated with any disease.
2) Recombinant AAV vectors can trigger long-term gene expression even in the absence of genome integration (at least in postmitotic tissues).
3) AVV vectors display only limited immunogenicity.
Due to these favorable properties, enthusiasm for gene therapy vectors based on adeno-associated virus (AAV) has steadily increased among researchers over the last few years. In fact, as of July 2015, 137 clinical trials using AAV have either been completed or are in progress. Moreover, the treatment of lipoprotein lipase deficiency with an AAV vector has been approved for clinical use in Europe, which is the first approval of gene therapeutic treatment in the Western World.
The research in our lab is focused on four areas:
1) The design of AAV variants with tissue and cell-specific tropism and increased resistance to pre-existing antibodies against the common AAV serotypes.
2) The development of AAV alternative approaches to overcome the hurdle that pre-existing anti-AAV antibodies pose to allow the use of AAV gene therapy for the broadest possible population segment.
3) To understand the basic biology of wild-type and recombinant AAV, especially as it relates to endocytosis and intracellular trafficking.
Physicians and scientists on the faculty of the Icahn School of Medicine at Mount Sinai often interact with pharmaceutical, device and biotechnology companies to improve patient care, develop new therapies and achieve scientific breakthroughs. In order to promote an ethical and transparent environment for conducting research, providing clinical care and teaching, Mount Sinai requires that salaried faculty inform the School of their relationships with such companies.
Below are financial relationships with industry reported by Dr. Weber during 2018 and/or 2019. Please note that this information may differ from information posted on corporate sites due to timing or classification differences.
- EMD Chemicals, Inc.
Mount Sinai's faculty policies relating to faculty collaboration with industry are posted on our website. Patients may wish to ask their physician about the activities they perform for companies.
Physicians who provide services at hospitals and facilities in the Mount Sinai Health System might not participate in the same health plans as those Mount Sinai hospitals and facilities (even if the physicians are employed or contracted by those hospitals or facilities).
Information regarding insurance participation and billing by this physician may be found on this page, and can also be obtained by contacting this provider directly. Because physicians insurance participation can change, the insurance information on this page may not always be up-to-date. Please contact this physician directly to obtain the most up-to-date insurance information.
Insurance and health plan networks that the various Mount Sinai Health System hospitals and facilities participate in can be found on the Mount Sinai Health System website.