Sentences with phrase «including gene therapy trials»

One early success story is cystic fibrosis: since the gene was identified in 1989, exceptional progress has been made in devising new experimental treatments, including gene therapy trials.

Not exact matches

There are now more than 100 gene therapy clinical trials aimed toward cancer, genetic diseases (such as ADA deficiency, cystic fibrosis and hemophilia A), infectious diseases (including AIDS) and autoimmune diseases (such as rheumatoid arthritis).

Women were not included in the first trial of gene therapy, which was mainly concerned with proving the safety of the technique.

His story inspired a book, The Forever Fix, which heralded the LCA trials as part of the wave of successes for gene therapy, which was recovering from several setbacks — including the death of a patient and cancer in others — in the late 1990s and early 2000s.

The current U.K. trial is again using fats, or lipids, but the DNA includes a segment called a promoter that should make gene expression last longer, says Eric Alton of Imperial College London, who heads the UK Cystic Fibrosis Gene Therapy Consortgene expression last longer, says Eric Alton of Imperial College London, who heads the UK Cystic Fibrosis Gene Therapy ConsortGene Therapy Consortium.

Other blood disorders that have shown significant benefit from targeted gene therapy in small trials include hemophilia (specifically, factor IX deficiency), severe beta - thalassemia (deficiency for the adult beta - globin gene) and leukemia, where the patient's immune cells were treated to enable them to recognize cancer cells and destroy them.

The vector is a self - inactivating lentivirus — a member of a family of viruses that can insert genes into mammalian cells and drive expression of those genes — that has been engineered to avoid triggering the development of leukemia, a complication seen in previous gene therapy trials for immunodeficiency syndromes, including WAS.

Colleen Dansereau, RN, MSN, CPN Program Manager, Gene Therapy Program As manager of the Gene Therapy Program, Colleen administratively manages the CAR T - cell therapy program as well as all administrative responsibility for all of the gene therapy clinical trials that take place at Dana - Farber / Boston Children's, for conditions including sickle cell disease, Wiskott - Aldrich Syndrome (WAS), and chronic granulomatous disease (CGene Therapy Program As manager of the Gene Therapy Program, Colleen administratively manages the CAR T - cell therapy program as well as all administrative responsibility for all of the gene therapy clinical trials that take place at Dana - Farber / Boston Children's, for conditions including sickle cell disease, Wiskott - Aldrich Syndrome (WAS), and chronic granulomatous diseaseTherapy Program As manager of the Gene Therapy Program, Colleen administratively manages the CAR T - cell therapy program as well as all administrative responsibility for all of the gene therapy clinical trials that take place at Dana - Farber / Boston Children's, for conditions including sickle cell disease, Wiskott - Aldrich Syndrome (WAS), and chronic granulomatous disease (CGene Therapy Program, Colleen administratively manages the CAR T - cell therapy program as well as all administrative responsibility for all of the gene therapy clinical trials that take place at Dana - Farber / Boston Children's, for conditions including sickle cell disease, Wiskott - Aldrich Syndrome (WAS), and chronic granulomatous diseaseTherapy Program, Colleen administratively manages the CAR T - cell therapy program as well as all administrative responsibility for all of the gene therapy clinical trials that take place at Dana - Farber / Boston Children's, for conditions including sickle cell disease, Wiskott - Aldrich Syndrome (WAS), and chronic granulomatous diseasetherapy program as well as all administrative responsibility for all of the gene therapy clinical trials that take place at Dana - Farber / Boston Children's, for conditions including sickle cell disease, Wiskott - Aldrich Syndrome (WAS), and chronic granulomatous disease (Cgene therapy clinical trials that take place at Dana - Farber / Boston Children's, for conditions including sickle cell disease, Wiskott - Aldrich Syndrome (WAS), and chronic granulomatous diseasetherapy clinical trials that take place at Dana - Farber / Boston Children's, for conditions including sickle cell disease, Wiskott - Aldrich Syndrome (WAS), and chronic granulomatous disease (CGD).

Weill Cornell is the birthplace of many medical advances — including the development of the Pap test for cervical cancer, the synthesis of penicillin, the first successful embryo - biopsy pregnancy and birth in the U.S., the first clinical trial of gene therapy for Parkinson's disease, and most recently, the world's first successful use of deep brain stimulation to treat a minimally conscious brain - injured patient.

Dr. Azad is a national leader in GI cancer, including serving as a member of the national NCI Colon Cancer Task Force and the NCI MATCH (Molecular Analysis for Therapy Choice) Agents and Genes Working Group, the largest trial of personalized medicine in the country.

Dr. Jerry Mendell of Nationwide Children's Hospital recently joined us for a webinar to discuss his upcoming gene therapy trial, including trial design, inclusion / exclusion criteria, and timelines.

Lysogene's MPS IIIA gene therapy clinical trial strategy includes a partnership with the MPS patient networks to ensure families are well informed about our development program and are able to make informed choices concerning the best treatment strategy for their children.

Treatment can include surgery to excise the mesothelial and surrounding tissue, chemotherapy, drug regimens, drug trials, gene therapies, radiation, and draining of fluid followed by the injection of medication.

New treatments are being tested to tackle the crippling difficulties of vertigo, including a clinical trial of prosthetic ear implants and ear gene therapy, with initial work revealing novel aspects of brain anatomy linked to balance which could be used as targets for future treatments.