Gene Repair

It is nice to see so many possible avenues being investigated to treat thalassemia and other blood disorders

Clifford J. Steer, M.D.
Professor







Education: M.D. University of Minnesota, 1974


 
Areas of Research Strength: Membranes and receptors; Cell interactions; Gene expression; Gene Therapy; Apoptosis
Research Techniques: Light, confocal and electron microscopy; Immunostaining and morphometric analysis; FACS analysis; Northern, western and Southern blotting; Isolation of nuclei, polysomes and mitochondria; Run-on transcription, in vitro RNA decay and RNase protection; Electrophoretic mobility gel shifts; Microarrrays; Tissue culture both primary and cell lines; Reporter gene assays - luciferase and CAT; PCR, RT-PCR, quantitative PCR and RT-PCR, real-time PCR; Apoptosis detection: TUNEL, annexin, mitochondrial and caspase assays; Plasmid and transgene construction using the Sleeping Beauty transposon system; Nonviral transgene and oligonucleotide delivery for gene augmentation and repair; Biochemical assays for gene activity - specific to model system being studied; Protein production and purification
Research Interests:
The Steer laboratory is involved in two major areas of research. In the first, the lab has over the last six years developed a novel gene therapy that involves the precise repair of genetic defects in cells. The lab has concentrated on the genetic repair of a variety of diseases, including hemophilia, sickle cell disease, Crigler-Najjar syndrome type I, ornithine transcarbamylase deficiency, ß-thalassemia, von Willebrand’s disease and certain neurodegenerative disorders like Huntington’s disease. In fact, Steer has successfully treated several accurate animal models for these human disorders. The technology of gene repair is remarkable for its broad application. The ability to now correct a genetic sequence in combination with the knowledge from the human genome project creates a remarkable vista of potential therapeutic clinical studies. The correction of a precise genetic defect allows the gene to be endogenously regulated without the potential problems associated with viral vectors. It may provide us with the first cures to diseases such as sickle cell and hemophilia. The Steer lab has successfully elucidated the basic science and will now apply the technology as therapy for human disease. In addition, the lab has also developed a more traditional type of gene therapy for diseases that are not candidates for genetic repair. However, while it is just as powerful as any traditional approach, it does not require potentially harmful viral vectors. The future of gene therapy is non-viral and that is the focus of our research as it applies to human therapy.

http://www.gcd.umn.edu/html/faculty_pages/steer.html

Yes so many research going on....

Congratulations for all in thalassemia world!!!

Thanks Dore - I hope that a solution will be found soon.  I hope also that the positive implications will be good for related blood disorders as well. 

Sharmin

Does anyone know how close we really are to actually having any of these cures.

The trial in the US will begin soon. It will take years to perfect and get approved. I think we will see many other advancements in treatment before a gene therapy cure is available. There is research into many different approaches to treating thal, such as a drug in stage 2 trials that will stimulate the production of fetal hemoglobin in a significant way, hopefully giving a 3-4 point rise in Hb. There is research into preventing iron absorption, correcting ineffective bone marrow activity and basically re-regulating various levels in the body that get far off balance in thals. All of there things will help to improve the lives of thals. When I have been able to attend conferences, I have been amazed at the many different areas of research going on regarding thalassemia. I see a future less dependent on transfusion and chelation than today, even without a cure.