March 22, 2021
Thanks to developments in precision medicine, some adult cancers are now treated with designer drugs that target the genetic mutations that caused them. But most children with cancer have not reaped the same benefits. Unlike adult cancers, childhood cancers carry few genetic mutations. And the mutations these tumors do have are typically harder to make drugs against.
March 8, 2021
Fionn Mulrooney, a cheerful 11-month-old, in Plymouth, Massachusetts, has no idea he has a life-threatening genetic disease. Nor does he seem fazed by the daily subcutaneous injections his parents have learned how to give him. And little does he know that cells from his bone marrow are helping scientists develop an innovative gene-editing approach that could someday correct his disease, known as severe congenital neutropenia or SCN.
December 22, 2020
As early as the 1960s, scientists speculated that DNA sequences could be introduced into patients’ cells to cure genetic disorders. In the early 1980s, David Williams, MD, and David Nathan, MD, at Boston Children’s Hospital published the first paper showing one could use a virus to insert genes into blood-forming stem cells. In 2003, the Human Genome Project wrapped up, giving us a complete blueprint of our DNA. In the past decade, gene therapy has become a reality for multiple diseases, especially those caused by mutations in a single gene.
February 1, 2019
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.
December 18, 2018
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.
December 3, 2018
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.
May 23, 2018
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.
December 7, 2017
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.
September 18, 2017
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.
August 31, 2017
Dana-Farber/Boston Children's is actively preparing to offer the novel treatment.