Gene Therapy -

Institute Structure

Research laboratories – include ten independent research groups, each directed by a Principal Investigator. These laboratories are conducting in vitro and in vivo studies, including the development of animal models and the assessment of platform technologies in each and every project.

The Hadassah hESCs cell Center site

National-Hadassah Gene Therapy Facility – produces molecular therapeutics, including proteins, DNA, viral vectors and cell therapeutics, all meeting cGMP guidelines of the EMA and FDA. The facility can accommodate three lines of biological therapeutics simultaneously. Once a project achieves a reasonable maturity, including proven potential in an animal model, it will advance towards the production step in the cGMP laboratory.

GMP laboratory– The main objective of the GMP Production Facility is the production of vectors and cells for use in human gene therapy clinical trials, primarily vectors for use in Phase I/II the trials. 

Clinical Research Ward: When the production process is completed, and the investigator's new drug file is approved, the product enters the clinical stage. The Clinical Research Ward is currently conducting a number of phase I and II clinical trials. The facility also supports other research groups both at the Hadassah medical center and elsewhere.

Agenda

The research agenda for our basic research facilities has been based on our view of possible obstacles which may be slowing down progress toward implementation of gene, cell and molecular therapy at the bedside.

We have decided to employ two independent, though interactive, research strategies concomitantly: The first is to apply currently available methods to resolve clearly defined research problems, or to develop new technologies essential to overcome them; the second is to conduct basic research in scientific fields related to gene therapy, such as immunology, virology, cell biology and biochemistry.

The following are issues currently addressed by our basic research facility:

- Gene delivery systems for specific tissues and organs; genomic targeting

- Low immununogenicity gene delivery vehicles

- Methods for enhancing or suppressing the immune response against transgenes or viral delivery systems

- The cellular environment in which the new gene is to be expressed, including interactions between proteins and the balance between degradation and production of proteins.

- Mechanisms used by gene delivery systems for entering cells. Further elucidation of the viral and cellular components i.e. ligands and receptors involved in binding, fusion, nuclear entrance and expression of the transgene

- Methods for site specific gene sequence correction.

- Animal models for human diseases

- Cellular trafficking machinery as a tool for nuclear entry and export

- Criteria for the selection of patients for each gene therapy project, based on genetic data and other information of the specific patient

- Controlled expression systems

- Cell therapy as a means of gene therapy

- Training of Ph.D. and M.Sc. students

For details on selected

Scientific Activities

under way at the Institute click here.