What is Gene Therapy?
Gene therapy is a type of medical treatment that involves replacing or modifying a person's genes in order to treat a disease or condition. In some cases, this involves introducing healthy copies of a gene into a person's cells to correct a genetic defect.
General Information About an IRB’s Role in Research
An Institutional Review Board (IRB) will review a research study to make sure that the researchers are conducting the study in a way that is safe for the participants and that the participants are being treated fairly and with respect.
An IRB will also review the study to make sure that the researchers are following all relevant laws and regulations. Overall, the role of an IRB is to ensure that research involving human subjects is ethical and conducted in a responsible manner.
What is a Natural History Study?
A natural history study is a type of research study that involves observing and collecting data on the natural progression of a particular condition or disease over a period of time. Natural history studies are often used to understand the early stages of a disease, how it progresses, and how it responds to different treatments.
What is a Biobank/Biorepository Study?
Biobanking refers to the collection, processing, preservation, and distribution of biological samples, such as blood or tissue, for use in research. Biobanks play an important role in supporting scientific research by providing researchers with access to high-quality biological samples that can be used to study a wide range of topics.
What are gene therapy clinical trials?
Clinical trials are research studies that involve people. These studies are done to find out if new medical treatments, such as drugs or medical devices, are safe and effective. Clinical trials are an important step in the development of new treatments, as they provide researchers with the information they need to determine whether a treatment is safe and effective for use in humans.
American Society of Gene & Cell Therapy (ASGCT) Patient Education
ASGCT is the primary professional membership organization for gene and cell therapy. The mission of ASGCT is to advance knowledge, awareness, and education leading to the discovery and clinical application of genetic and cellular therapies to alleviate human disease.
National Organization for Rare Diseases (NORD)
NORD is a patient advocacy organization dedicated to individuals with rare diseases and the organizations that serve them. NORD, along with its more than 300 patient organization members, is committed to the identification, treatment, and cure of rare disorders through programs of education, advocacy, research, and patient services.
National Institutes of Health (NIH)
