Sentences with phrase «create gene therapy treatments»

There a team has been working to create gene therapy treatments for GM1 in cats.

Not exact matches

The SMA model pig was created using a gene therapy approach by knocking down the levels of pig SMN, followed by treatment with human SMN at early and late time points.

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.

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.

«With our enhanced method of direct cardiac reprogramming, we hope to combine gene therapy with drugs to create better treatments for patients suffering from this devastating disease.»

The Company's transformative gene repair technology presents a promising opportunity to create novel therapies that address the significant unmet need in the treatment of serious genetic neuromuscular diseases.

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.

AGTC plans to use the net proceeds of the offering to fund preclinical investigation and Phase 1/2 trials of potential product candidates for treatment of wet AMD; to expand its manufacturing capabilities and create a pilot manufacturing group; to in - license, acquire or invest in complementary gene therapy, technologies, products or assets; and for working capital and other general corporate purposes.