The study of gene therapy

Here, her mom, Lonnel, assists Kate with an aerosol machine.

The study of gene therapy

The study of gene therapy

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WEDNESDAY, March 13, — A gene therapy that protects cartilage could one day treat osteoarthritis, a condition affecting more than half the aging population, according a study published in the journal Science Translational Medicine.

Gene therapy for sickle cell anemia

Osteoarthritis OA is a natural response to the breakdown of joint cartilage — a spongy material that keeps bones from rubbing against each other. As cartilage thins because of age or due to an injury, that barrier breaks, and the friction causes pain.

The only cure is joint replacement surgery, which can result in surgical complications. This gene therapy slows the development of the disease in mice.

Lee and his team targeted a specific gene within joint cartilage called proteoglycan or PRG4. This gene is responsible for producing lubricin, a protein that lubricates the joint and protects cartilage from the effects of old age.

Vision Loss in Batten Disease Prevented by Gene Therapy in Mouse Study

The scientists injected joint damaged mice with PRG4 encased inside a virus, which spread the gene throughout the area and stimulated lubricin production.

The extra lubricin significantly slowed the development of OA with no perceivable side effects. Researchers came up with the idea while studying skeletal disorders caused by rare genetic defects. You have to be guarded in your enthusiasm.

And even if the therapy was eventually approved for human use, Lee says, it could only slow the loss of cartilage, not regrow it. The researchers say that the industry must come together and share advances in technology in order to create a comprehensive treatment plan.New research by Dr.

Donald Kohn of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA could make blood stem cell gene therapy .

Latest from CBS News

Study Note 2: The technique of somatic gene therapy The technique of somatic gene therapy involves inserting a normal gene into the appropriate cells of an individual affected with a genetic disease, thereby permanently correcting the disorder.

Nov 13,  · Gene therapy is under study to determine whether it could be used to treat disease.

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Current research is evaluating the safety of gene therapy; future studies will test whether it is an effective treatment option. Nov 08,  · This is the story of how an experimental gene therapy saved a little boy's life, giving hope to the hundreds of thousands of families affected by EB worldwide.

The study . WEDNESDAY, March 13, — A gene therapy that protects cartilage could one day treat osteoarthritis, a condition affecting more than half the aging population, according a study published in.

The first patient has been treated in a phase 2b dose-confirmation study of AMT, an investigational gene therapy for the treatment of patients with severe and moderately severe hemophilia B.

Investigational Breast Cancer Gene Therapy Could Improve Reconstruction, Study Suggests