Related Questions

Is there any research being done to start gene therapy for scheuermann disease?

Check this site. Not sure, but www. Clinicaltrials. Gov has information on any studies that would be looking into this. Look at this link: http://clinicaltrials. Gov/ct2/results? Term=calve+disease (i just put in calve disease as a search term, to get broader results). Good luck!

How do I know when will gene therapy become a reality for orphan diseases (single gene disorders)?

Glybera. The first gene therapy has now been approved in europe for an orphan disease. Several hundred therapies are currently in human clinical trials. So, I would say gene therapy is now a reality and the next 5 years will see a number of therapies approved.

What disease is gene therapy is used to treat?

Gene therapy. Treating certain congenital enzyme deficiency metabolic disorders. Looks promising in hemophilia where research going. Also lot of interest in cancers and treating infectious diseases.
Selected. Gene therapy describes an approach whereby genetic technology will be used to treat many conditions. The easiest use of gene therapy is to replace a faulty gene. An example is glybera which is approved in europe to treat lipoprotein lipase deficienct, a rare genetic disorder.

Hello docs, is gene therapy the cure for genetic disease?

Could be. Right now it is too early to say. It's in early research phases. Also, it depends on which genetic disease. A disease caused by a single amino acid substitution may be easier to "cure" than one with multiple genetic loci and epigenetic and/or methylation effects.

Is gene therapy for maple syrup urine disease?

Not Yet. Msud is a very rare genetic d/o where one is unable to break down the Amino Acids (aa): leucine, isoleucine, & valine. The degree to which an individual might be affected varies. In the us it is diagnosed with neonatal screening which takes place when the baby is around 24 hrs old. Treatment is with dietary avoidance of these aa to spare brain damage and possible death.

Is there gene therapy for cystic fibrosis and health centrefor this disease in the us?

No and yes. Safe effective gene therapy for CF is a dream that we have yet to realize, but there are many excellent CF centers in the us. The CF foundation is an excellent source of information on these centers. The treatments for CF are improving, and expanding constantly. The life expectancy for patients with CF has steadily increased but is still limited by pulmonary disease.

Help please? What is gene therapy and how might it be used as a treatment for cancer or for genetic disorders?

Gene therapy 4future. Gene therapy is still experimental. In the future it may help treating a disorder by inserting a gene into patient’s cells instead of drugs or surgery. Although it is promising option for some diseases (some inherited disorders, some types of cancer,), there are still risks and it is still in studies to be sure it will be safe and effective. It is not tested for diseases that have no other cures.