Preliminary results from a landmark clinical trial suggest that CRISPR–Cas9 gene editing can be deployed directly into the body to treat disease. The study is the first to show that the technique can be safe and effective if the CRISPR–Cas9 components — in this case targeting a protein that is made mainly in the liver …
Landmark CRISPR Trial Shows Promise Against Deadly Disease Read More »
The gene editor CRISPR excels at fixing disease mutations in lab-grown cells. But using CRISPR to treat most people with genetic disorders requires clearing an enormous hurdle: getting the molecular scissors into the body and having it slice DNA in the tissues where it’s needed. Now, in a medical first, researchers have injected a CRISPR …
CRISPR Injected Into the Blood Treats a Genetic Disease for First Time Read More »
People with hereditary transthyretin (ATTR) amyloidosis have a mutation in the transthyretin (TTR) gene, which means they produce an abnormal protein that gradually builds up in the heart and nerves. Symptoms include numbness in the hands and feet, loss of control of the bowel and bladder and immobility. The condition gets progressively worse and is …
Gene Editing Therapy Paves Way for Revolution in Treatment of Genetic Disorders Read More »
Researchers from Intellia Therapeutics, Regeneron Pharmaceuticals, and clinical partners have published the first-ever clinical data that support the safety and efficacy of in vivo CRISPR genome editing in humans. The team of researchers based in the U.S., the U.K., and New Zealand showed in a study that the companies’ lead in vivo genome editing candidate …
The treatment uses CRISPR/Cas9 editing technology, a technique derived from bacteria that allows researchers to edit the genome of a cell by specifying regions of DNA to be added, removed, or edited. NTLA-2001 is designed to eliminate the TTR gene from the DNA of liver cells, which would reduce TTR production significantly and prevent the …
UK Approves Phase 1 Trial of Potential Gene Editing Therapy, NTLA-2001 Read More »
What Is a Clinical Study? A clinical study involves research using human volunteers (also called participants) that is intended to add to medical knowledge. There are two main types of clinical studies: • Interventional studies • Observational studies Clinical Trials In a clinical trial, participants receive specific interventions according to the research plan or protocol …
The term informed consent is mistakenly viewed as the same as getting a research participant’s signature on the consent form. FDA believes that obtaining a research participant’s verbal or written informed consent is only part of the process. Informed Consent Involves Providing a Potential Participant With: Adequate information to allow for an informed decision about …
You may ask yourself, “Why should I try something that researchers are not sure will work?” That is a good question. Being part of a clinical trial may have risks, but it may also have benefits. Past clinical trial history has led many to hesitate to sign up for research. However, today there are strict …
Clinical trials are research studies that include human volunteers. Before they start a clinical trial, doctors must prove there is a chance that the new treatment will work better than the one available now. They do research until they can prove this. For example, they might test the treatment on laboratory animals. They do this …
This glossary will help you understand words and phrases frequently used on ClinicalTrials.gov. Many of these words are also used by clinical researchers and others in the same or a similar manner. But the definitions below are provided to explain content on ClinicalTrials.gov only. Accepts Healthy Volunteers A type of eligibility criteria that indicates whether …