Currently, there are no on-going gene therapy or stem cell therapy studies for patients which chromosome abnormalities such as a deletion or duplication. In the United States, gene therapy is currently available only in a research setting. It is our understanding that the U.S. Food and Drug Administration (FDA) has not yet approved any gene therapy products for sale in the United States.
Some of the diseases for which gene and stem cell therapies are is being investigated on a research basis include:
You can find more information about gene therapy from Genetics Home Reference, the U.S National Library of Medicine's Web site for consumer information about genetic conditions and their related genes.
The University of Utah's Genetic Science Learning Center provides comprehensive resources on this topic including discussions on the challenges in gene therapy and new strategies to gene therapy.
Although no studies involving gene and stem cell therapy for chromosomal disorders are listed at this time, you can check the website ClinicalTrials.gov often for updates. The U.S. National Institutes of Health, through the National Library of Medicine, developed ClinicalTrials.gov to provide patients, family members, and members of the public with current information on clinical research studies.
You can also contact the Patient Recruitment and Public Liaison (PRPL) Office at the National Institutes of Health (NIH). We recommend calling 1-800-411-1222 to speak with a specialist, who can help you determine eligibility for any clinical trials. If you are located outside the United States, and would like to be contacted via telephone, you will need to contact PRPL and provide your telephone number in full, including area code and international dialing prefix.
Patient Recruitment and Public Liaison Office (PRPL)
NIH Clinical Center
Bethesda, Maryland 20892-2655
Web site: http://clinicalcenter.nih.gov/
Nonprofit support and advocacy groups bring together patients, families, medical professionals, and researchers. These groups often raise awareness, provide support, and develop patient-centered information. Many are the driving force behind research for better treatments and possible cures. They can direct people to research, resources, and services. Many groups also have experts who serve as medical advisors. Visit their website or contact them to learn about the services they offer. Inclusion on this list is not an endorsement by GARD. Suggest an organization to add.
These resources provide more information about this condition or associated symptoms. The in-depth resources contain medical and scientific language that may be hard to understand. You may want to review these resources with a medical professional.
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Can a chromosome 8p duplication be corrected? Is there any hope in experimental treatments such as gene therapy or stem cell manipulation? See answer