Sentences with phrase «thalassemia using»

Successful treatment of murine ß - thalassemia using in vivo selection of genetically - modified, drug resistant hematopoietic cells.

Not exact matches

For instance, a couple who each suffers from beta thalassemia might only have healthy children free from the inherited blood disorder if they were able to produce embryos in which the genetic defect was corrected using gene editing.

Then a team of Chinese researchers used that base editor to correct a mutation in human embryos that causes the blood disorder beta - thalassemia, reported September 23 in Protein & Cell (SN: 11/25/17, p. 7).

Cord - blood stem cells have been used since 1988 to treat a wide variety of leukemias, thalassemias and other primarily blood - related illnesses.

Other plans include using CRISPR to reverse blood disorders, such as sickle cell anemia and beta thalassemia, caused by mutations in the hemoglobin gene.

A Yale - led research team used a new gene editing strategy to correct mutations that cause thalassemia, a form of anemia.

To achieve gene editing in mice with thalassemia, professor of therapeutic radiology and of genetics Peter M. Glazer, M.D. and his co-authors developed an alternative approach using a novel combination of nanoparticles, synthetic pieces of DNA, and a simple IV injection.

When they tried correcting the mutations using CRISPR - Cas9, their success rate was one in four for β - thalassemia and two in two for favism, they report this month in Molecular Genetics and Genomics.

In contrast to Ciclopirox, approved for topical use, Deferiprone is FDA - and EMA - approved for systemic use (in certain thalassemia patients with iron overload).

Deferiprone, available for the past 20 years, is used in more than 60 countries to prevent death from blood transfusion - related complications during treatment of thalassemia, an inherited blood disorder in which the body makes an abnormal form of hemoglobin.

CRISPR / Cas9 could be used to develop therapies for humans for genetic blood diseases such as sickle cell or thalassemia, and this paper does not change that potential.

The Bone Marrow and Stem Cell Transplant Program is expanding the use of this technique for patients with solid tumors including neuroblastoma and brain tumors; a variety of high - risk hematologic diseases, such as thalassemia major and transfusion - dependent sickle cell disease; and other nonmalignant diseases.

In a recent study, Chinese researchers used CRISPR / Cas in human embryos to correct a mutation that causes the blood disease beta - thalassemia.

The BBC reports that gene therapy has been used successfully to treat a patient with severe β - thalassemia.

Seamless gene correction of β - thalassemia mutations in patient - specific iPSCs using CRISPR / Cas9 and piggyBac.