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Researchers at Dana-Farber/Boston Children's and UMass Medical School optimize gene editing for sickle cell disease and beta thalassemia

March 28, 2019
Researchers at Dana-Farber/Boston Children's Cancer and Blood Disorders Center and the University of Massachusetts Medical School have developed a strategy to treat two of the most common inherited blood diseases, sickle cell disease and beta thalassemia, applying CRISPR-Cas9 gene editing to patients' own blood stem cells.

Dana-Farber/Boston Children's Brian D. Crompton, MD, awarded research grant by the Go4theGoal Foundation

March 25, 2019
The Go4theGoal Foundation (G4TG) recently awarded Brian Crompton, MD, of the Dana-Farber/Boston Children's Cancer and Blood Disorders Center, a $50,000 research grant.

Scott Armstrong, MD, PhD, recognized by ISSCR for contributions to basic and translational research in hematological disorders

March 25, 2019
The International Society for Stem Cell Research (ISSCR) announced that Scott Armstrong, MD, PhD, is the recipient of its 2019 Tobias Award Lecture.

Transplants from any donor, without toxicity?

February 06, 2019
Pre-treatment with an antibody-drug combination avoids side effects in mice; could substitute for current toxic regimens using chemotherapy or radiation

Multi-center study led by Dana-Farber/Boston Children's contributes to FDA approval of a new treatment for acute lymphoblastic leukemia

February 04, 2019
Multi-center study led by Dana-Farber/Boston Children's contributes to FDA approval of a new treatment for acute lymphoblastic leukemia

Boston Children’s Hospital to receive $1.5M to initiate basic work to expand gene therapy treatment of sickle cell disease into developing countries

December 19, 2018
The research will focus on adapting gene therapy for use in low-resource areas with high rates of the disease.

CRISPR-Cas9 screen opens new targets for Ewing sarcoma, other childhood cancers

December 18, 2018
In this Vector report, research finds that drugs targeting the TP53 pathway can curb tumor cell proliferation in Ewing sarcoma.

New gene therapy strategy for sickle cell disease shows early promise in humans

December 01, 2018
A patient with a severe form of sickle cell disease is symptom-free after gene therapy knocks down BCL11A and restores fetal hemoglobin production.

Fishing for new leads in rare mucosal melanoma

November 01, 2018
Vector reports that zebrafish are helping scientists dissect intricate molecular pathways in cancer and discover new treatment approaches.
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