Sentences with phrase «as gene therapy treatments»

These human genes were also protective against alpha - synuclein - induced death, suggesting that they could be worth testing as gene therapy treatments for Parkinson's disease, Lu says.

«Both of the designed linker proteins may possibly be used in the future as a gene therapy treatment for congenital muscular dystrophy,» says Rüegg.

Not exact matches

The treatment, named Kymriah, was hailed by doctors and the life sciences community as a major advance in medicine and a boon to children and young adults with a certain form of leukemia (the group for whom the gene therapy is approved).

Luxturna is the first of a crop of treatments that target diseases caused by mutations in specific genes, and thus is referred to by many as the first gene therapy in the U.S.

So far, its trials have shown it can improve outcomes when used alongside other multiple myeloma drugs and that could offer it some insulation if the market gets disrupted by new treatment approaches, such as gene therapy.

Gene therapies promise to revolutionize the treatment of many diseases, including neurological diseases such as ALS.

These include the ability to bring new, innovative products to the market; progress in oncology, such as the approval of Genentech's drug Avastin for breast cancer and advances in the use of gene therapy, despite some setbacks; continuing progress in research on stem cells; the emergence of treatments for previously untreated diseases; and solutions for food and fuel shortages, such as biocrops and biofuels.

Tsigelny added: «The clinical treatments that do not take into consideration the specific mutations in genes, but rather treat all mutations within a specific gene such as EGFR as the same, are a «hit - or - miss» game because they are based solely on observational data on how other patients previously have reacted to drug therapies.»

Kohn's gene therapy approach using HSC from patient's own blood is a revolutionary alternative to current SCD treatments as it creates a self - renewing normal blood cell by inserting a gene that has anti-sickling properties into HSC.

Under the proposed law, gene therapy will be approved only for the treatment of people with genetic diseases such as cystic fibrosis and will not be allowed in germ cells, where genetic alterations would be passed on to the next generation.

As a new generation of gene therapy clinical trials shows promise to cure or halt the progression of several rare diseases, the time has come to explore ways to pay for the cutting edge treatments, a pediatric hematologist - oncologist from Dana - Farber / Boston Children's Cancer and Blood Disorders Center argues in a commentary published by the journal Science.

And if, as he expects, gene therapy replaces these treatments in the next decade, patients will in any case not have lifetime exposure to PEG.

Reference materials are critical to properly evaluate the next - generation of gene sequencing and genetic testing methods that will increase the reliability and effectiveness of precision medicine (also known as «personalized medicine»), in which a person's genetic profile is used to create treatments and therapies unique to that individual.

The research, part of a phase I clinical trial to test the safety of the treatment, was published as a letter to the editor in The New England Journal of Medicine earlier this week and will be in the September issue of Human Gene Therapy.

«RNAi therapies are a unique approach to cancer treatment as they have the potential to «turn off» the genes» coding for proteins involved in cancer cell division,» said Ramesh K. Ramanathan, M.D., medical director of the Virginia G. Piper Cancer Center Clinical Trials Program at Scottsdale Healthcare and deputy director of the Clinical Translational Research Division of the Translational Genomics Research Institute (TGen) in Phoenix, Ariz. «Using a lipid nanoparticle, the RNAi drug can be delivered to a cancer cell to block the expression of specific proteins involved in tumor growth.»

A «miracle» gene therapy treatment for children suffering from the fatal «bubble boy» disease has been halted in France, after one of the patients developed leukaemia as a direct consequence of the treatment.

At a time when gene therapy has been revived as a potent form of cancer treatment, a new approach would use nanoparticles, rather than viruses, to deliver strands of DNA or RNA to tumors.

Hence, scientists have begun studying BRCA - targeted gene therapy as an effective treatment for breast cancer.

This specificity in treatment fits with an emerging approach in cancer treatment nationwide, known as personalized medicine, in which the therapies for each patient are selected based on the genes altered in their tumors.

While still primarily performed as part of research studies, gene therapy is becoming more widely adopted for clinical treatment of some conditions.

Gene - editing tool could replace antiretroviral therapy as treatment for HIV.

For those patients without fully matched donors, researchers are working to devise better treatments, such as gene therapy.

A new animal study involving virally - delivered gene therapies has indicated that high treatment doses might not be as safe as previous human trials have suggested

The platform serves as a potential way to help researchers quickly discover new carriers for a broad range of treatments, from small molecules to gene therapies.

Clinical research activities are focused on the implementation of experimental protocols based on gene or cell therapy for the treatment of genetic blood disorders such as ADA - SCID, Wiskott - Aldrich Syndrome, Metachromatic Leukodystrophy, Beta - thalassemia, Chronic granulomatous disease, Mucopolysaccharidosis Type I (Hurler).

In 2007, a 36 - year - old Chicago woman died three weeks after her knee was injected with genetically engineered viruses as part of an investigational treatment researching gene therapy's potential in easing the pain of rheumatoid arthritis.

«Our work in NPC1 mice may help lead to human clinical trials and eventually FDA approval for gene therapy as a treatment for NPC1 disease,» said Charles P. Venditti, M.D., Ph.D., senior investigator in the NHGRI Medical Genomics and Metabolic Genetics Branch.

Up to 50 million Americans are estimated to have an autoimmune condition, * and annual direct healthcare treatment costs for these patients total $ 100 billion.1 Nearly 100 different diseases have been classified as autoimmune in nature, with 40 more suspected.1 IFM's 2018 Annual International Conference (AIC), «Solving the Puzzle of Autoimmunity: The Interplay of Gut, Genes, and Environment,» focuses on the exploding research and therapies around the interconnected roles of intestinal permeability, genetic disruptors, and various environmental triggers that coalesce to create autoimmune disease.

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.