Categories: Uncategorized

CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia



Transfusion-dependent β-thalassemia (TDT) and sickle cell disease (SCD) are the most common monogenic diseases worldwide, with an annual diagnosis in approximately 60,000 patients with TDT and 300,000 patients with SCD. Both diseases are caused by mutations in the hemoglobin β subunit gene (HBB).…



Source link

sony

Share
Published by
sony

Recent Posts

Environmental Factor – August 2017: Remembering children’s health champion Herbert Needleman

Herbert Needleman, MD, a physician-scientist whose lead in consumer products was removed due to research…

1 month ago

Environmental Factor – August 2017: FARE awards recognize 17 NIEHS fellows for outstanding research

ਸਤਾਰਾਂ ਐਨਆਈਈਐਚਐਸ ਫੈਲੋਜ਼ ਨੂੰ 199 ਨੈਸ਼ਨਲ ਇੰਸਟੀਚਿ ofਟ ਆਫ਼ ਹੈਲਥ (ਐਨਆਈਐਚ) ਦੇ ਖੋਜਕਰਤਾਵਾਂ ਵਿੱਚ ਖੋਜ…

1 month ago

Extramural Papers of the Month

(ਹੋਰ ਪੜ੍ਹੋ) Source link

1 month ago

Environmental Factor – January 2021: Woychik shares leadership philosophy and goals

In this inaugural director’s column, I would like to share with you what makes me…

1 month ago

Induction of Fetal Hemoglobin by Gene Therapy

Shortly after birth, fetal hemoglobin is replaced by adult hemoglobin in red cells, a process…

1 month ago

Treatment by CRISPR-Cas9 Gene Editing — A Proof of Principle

As a tool of great promise for the treatment of inherited human diseases, the clustered…

1 month ago