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These Patients Had Sickle-Cell Disease. Experimental Therapies Might Have Cured Them.
Scientists have long known what causes sickle-cell disease and its devastating effects: a single mutation in one errant gene. But for decades, there has been only modest progress against an inherited condition that mainly afflicts people of African descent. With advances in gene therapy, that is quickly changing — so much so that scientists have begun to talk of a cure.
Read moreGates Foundation gives $1.5M to Children’s for sickle cell research
The Bill & Melinda Gates Foundation has donated $1.5 million to Boston Children’s Hospital to develop a gene therapy for sickle cell disease, with the goal of making the drug technology more widely available in regions of the world with high rates of the condition.
Read moreGene therapies could transform the treatment of sickle cell disease
The potential therapies being developed and tested for sickle cell manipulate genes in a variety of ways, giving researchers a multitude of options and reason for hope.
Read more“NOVA Wonders” spotlights Boston Children’s researchers and clinicians as they undertake a groundbreaking genetic therapy that’s bringing hope to families of children with ALD
The fifth installment in NOVA's 6-part series, "NOVA Wonders," which aired Wednesday, May 23rd, features David Williams, MD, Boston Children's Chief Science Officer, Chief of the Division of Hematology and Oncology, and principal investigator on the trial, with Christine Duncan, MD, co-lead author on the study and a pediatric hematologist/oncologist at the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, and Colleen Dansereau, MSN, RN, CPN, CPHON, Program Manager for Boston Children's Gene Therapy Program.
Read moreNew Gene Therapy Transplantation Technique Could Improve Treatment Of Neurodegenerative Diseases
MedicalXpress reports that a therapeutic technique to transplant blood-forming stem cells directly into the brain - a technique reported in Science Advances yesterday - could herald a revolution in our approach to treating central nervous system diseases and neurodegenerative disorders.
Read moreHyundai Hope On Wheels To Award $400,000 Research Grant To Dana-Farber Cancer Institute Of Boston In Honor Of National Childhood Cancer Awareness Month
On Sunday, September 24, 2017, Hyundai Hope On Wheels® will present Dana-Farber Cancer Institute with a total of $400,000 from the Hyundai Scholar Hope and Young Investigator grant.
Read moreLandmark moment for science as the FDA approves a gene therapy for the first time
Dana-Farber/Boston Children's is actively preparing to offer the novel treatment.
Read moreHow Precision Medicine Could Be A Lifesaver For Kids With Brain Cancer
A team of Dana-Farber scientists has released new research with an important message about precision medicine: Sequencing the genes of brain tumors in kids could point to treatments that target their genetic abnormalities and therefore have the best chance of being effective.
Read moreGene therapy trial gives Coopersville toddler second chance at life
Colton was born healthy almost two years ago, but his fraternal twin brother, Levi, was not. The Michigan toddler had severe combined immune deficiency – “bubble boy” disease – discovered through newborn screening, Fox 17 reports from Grand Rapids.
Read moreNew Chief Scientific Officer at Boston Children’s Hospital
Boston Children’s Hospital has established a new executive role to focus on research, the hospital announced. Dr. David Williams, who serves as chief of hematology and oncology and director of clinical and translational research at Boston Children’s Hospital, will serve as chief scientific officer.
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