Archive for gene therapy
A potential danger of CRISPR gene editing — and why base editing may be safer
Gene therapy using CRISPR/Cas9 gene editing is currently in clinical trials around the world for a variety of diseases, including various cancers, blood disorders, and metabolic disorders. It works by making cuts in DNA — both strands of the double helix — to insert or remove genes. But CRISPR carries a potential, previously undiscovered danger, ... Read More
Sickle cell disease, gene therapy, and blood cancers: Mysteries remain
Gene therapy trials for sickle cell disease have been showing great promise, even offering hope of a cure. But in early 2021, the trials ground to a halt after reports of blood malignancies in two people in a trial sponsored by bluebird bio. Investigations later concluded that the gene therapy delivery vectors were likely not ... Read More
Global services: Coordinating care for international families
In most years, more than 2,500 families living outside of the United States seek care at Boston Children’s Hospital. To help guide the process, the Global Services team — a group of medical and administrative professionals — manages all the details. Their mission is to help coordinate care between families, medical professionals in their home ... Read More
Nurses Week 2021: Integrating new therapies into nursing practice and patient care delivery
A revolution in pharmacological gene therapy is underway, as indicated by a significant acceleration in the creation of new therapies to treat genetic disorders at the molecular level. Boston Children’s Hospital Nursing and extended team members, in collaboration with hospital researchers and scientists, biotech, and biopharma partners, have developed a set of specific processes to ... Read More
Teaming up to fast track a novel gene therapy for Diamond-Blackfan anemia
Looking at Lindsay Krieg, it’s impossible to tell she has spent her entire 29 years in an out of hospitals managing a rare blood disorder. “It’s routine for me,” she says. “It’s all I’ve ever known.” Diamond-Blackfan anemia (DBA) prevents Lindsay’s bone marrow from producing the red blood cells essential for carrying oxygen from her ... Read More
Matthew, the ‘wee marvel’: One of the first ALD gene therapy recipients
When the Elliott brothers are asked how many siblings they have, they always say, “four.” It’s a way of honoring the memory of their eldest brother, Marc, who died more than 10 years ago of adrenoleukodystrophy (ALD). ALD is a degenerative condition that destroys the protective sheath surrounding the brain’s neurons. Gradually, as the disease ... Read More
After decades of evolution, gene therapy arrives
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 ... Read More
A rebirth in Boston: Gene therapy turns 10
Lea la versión en español. Dec. 17 marks a decade since Agustín Cáceres was “renacido” — reborn. That’s how his parents, Alberto and Marcela, describe the day their son received his new gene. Born in 2010 with X-linked severe combined immunodeficiency (SCID-X1), Agustín spent the first few months of his life in isolation, at home ... Read More
Un renacimiento en Boston: la terapia genética cumple 10 años
Read the English version. El 17 de diciembre se cumple una década desde que Agustín Cáceres “renació”. Así describen sus padres, Alberto y Marcela, el día en que su hijo recibió su nuevo gen. Nacido en 2010 con inmunodeficiencia combinada grave ligada al cromosoma X (SCID-X1), Agustín pasó los primeros meses de su vida en ... Read More
Hemophilia A gene therapy paves the way for a life with less pain
Matthew Harrington, a 35-year-old oil-field worker from Douglas, Wyoming, knows pain. Born with hemophilia A, a rare condition in which the blood doesn’t clot properly, Matt’s body — both inside and outside — has difficulty stopping bleeding. He has what’s considered the most severe form of hemophilia, characterized by frequent bleeding episodes, particularly in the ... Read More