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January 05, 2010

Geron Stem Cells to Treat Brain Cancer, Stem Cell Facelift and Gene Therapy Updates

1. Stem cell facelift are available in India and are somewhat like Botox but use stem cells instead of poison

Stem cell facelift is carried out under local anaesthesia and fat is harvested from the patient's body manually using a syringe, usually from the lower abdomen where there is a plentiful supply. The fat is then processed through a machine that cleans the cells, which the practitioner injects with the fat and stem cells around the muscles, into the soft skin tissues in the face. This method is not only for face. One can rejuvenate bony or aged hands as well.

Stem cells have the quality of developing new types of cells. When they are transplanted to the facial skin, the skin will naturally be younger-looking because it is renewing at a much faster rate," she explained.

Agarwal claimed the procedure has no side effects. "There is no risk of damaging facial nerves, arteries or veins. It causes no allergies as it uses your own donated fat and recovery time is also less. Rs.50,000-65,000 (USD 1,084 to 1250) for the procedure, which should be carried out by a qualified cosmetic surgeon.

2.
Geron said its drug candidate imetelstat specifically inhibited the activity of the brain cancer, glioblastoma. The stem-cell technology inhibited tumor activity and reduced tumor size in a laboratory study focusing on the most common form of brain cancer. In the study, tumors were implanted into mice and imetelstat inhibited 60 percent to 70 percent of the tumors in up to five days. Geron plans to move the drug candidate into several Phase II clinical trials later in 2010.







3. At the University of Pennsylvania, Dr. Carl June and colleagues have used zinc finger gene therapy to disrupt a gene in patients’ T cells, the type attacked by the AIDS virus. They have then infused those cells back into the body. A clinical trial is now under way to see if the treated cells will reconstitute a patient’s immune system and defeat the virus.

In principle, the zinc finger approach should work on almost any site on any chromosome of any plant or animal. If so, it would provide a general method for generating new crop plants, treating many human diseases, and even making inheritable changes in human sperm or eggs, should such interventions ever be regarded as ethically justifiable. Zinc fingers are essential components of proteins used by living cells to turn genes on and off.

Daniel F. Voytas, a plant geneticist at the University of Minnesota, said the zinc finger technique would allow breeders to change the oil composition of any plant, the types of carbohydrates produced or the way carbon dioxide is captured. “We can go in and make any change we want to any plant species,” Dr. Voytas said.

Zinc fingers can also be used for “trait stacking,” the positioning of several beneficial genes at a single site. This avoids heavy regulatory costs because genetically altered plants must be tested for safety for each site that is modified.

The zinc finger technology has taken many years to prepare because of the difficulty of designing the fingers and also of preventing them from cutting the genome in the wrong places. Only a handful of laboratories are currently using the technique, but proponents expect to see rapid growth.

The Zinc Finger Consortium, founded by Dr. Joung and Dr. Voytas, makes the method available free, and researchers need only pay for materials. But there are some 200 steps in Dr. Joung’s recipe for making zinc fingers, and it takes time and dedication to do them all correctly.

Sigma-Aldrich’s charge for a zinc finger protein that cuts the genome at the site of your choice is $39,000, with a discount for academic researchers. Zinc fingers that cut well-known human genes cost $12,000.

Zinc fingers could be the gift that stem cell researchers have been waiting for. Stem cells taken from a patient may need to be genetically corrected before use, but until now there had been no way of doing so. Zinc finger scissors are so efficient that only 5 to 10 embryos need be treated to get one with the desired result





Sangamo BioSciences, Inc. is the worldwide leader in the design and development of engineered zinc finger DNA-binding proteins (ZFPs) for gene regulation and gene modification.

Sangamo is focused on the research and commercial development of engineered DNA-binding proteins for the regulation of gene expression and for gene modification. The basis of our proprietary technology is the engineering of a naturally occurring class of transcription factors, zinc finger DNA-binding proteins (ZFPs). When linked to functional domains that normally activate or repress gene expression, we create ZFP transcription factors (ZFP TFs) capable of turning genes on or off. ZFPs can also be linked to nuclease domains to create zinc finger nucleases (ZFNs) which enable precise gene-editing operations in cells. Engineered ZFNs can modify a cell's DNA at a preselected location facilitating correction or disruption of a specific gene or the targeted addition of new DNA sequence.

A New Class of Therapeutics
Our primary mission is to develop ZFP TF and ZFNs as ZFP Therapeutics™; ZFP TFs designed to directly regulate disease-related genes and ZFNs to disrupt or correct therapeutically relevant genes.



4. Biomarkers predict gene therapy efficiency and could be used to guide what gene therapy treatment should be used for each individual. This would lead to personalized gene therapy treatment.

Goodwin explains that using biomarkers to predict the efficacy of a treatment is critically important for patients with short life expectancies. "If we can better predict which treatment will be the most effective," he says, "we can apply that therapy as quickly as possible. The very low levels of side effects and toxicity seen in this study are also exciting."

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