Archive for gene therapy
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 about Nurses Week 2021: Integrating new therapies into nursing practice and patient care delivery
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 about Teaming up to fast track a novel gene therapy for Diamond-Blackfan anemia
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 about After decades of evolution, gene therapy arrives
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 about A rebirth in Boston: Gene therapy turns 10
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 about Un renacimiento en Boston: la terapia genética cumple 10 años
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 about Hemophilia A gene therapy paves the way for a life with less pain
Gene therapy with a new base editing technique restores hearing in mice
Using a new genetic engineering technique, known as base editing, researchers from Boston Children’s Hospital and the Broad Institute of MIT and Harvard, have restored hearing in mice with a known recessive genetic mutation. Key takeaways· This is the first example of repairing a recessive gene mutation.· Repairing a single mutation in the Tmc1 gene restored partial hearing in ... Read More about Gene therapy with a new base editing technique restores hearing in mice
Boston Children’s nurses: Setting the standard for innovation
As clinicians at Boston Children’s Hospital continue to pioneer new therapies for both kids and adults, nurses play a critical role in bringing those treatments to families. To make sure groundbreaking treatments like gene therapies are integrated into both nursing practice and patient care, Boston Children’s nurses have developed a series of processes. When ... Read More about Boston Children’s nurses: Setting the standard for innovation
Gene therapy reverses heart failure in mouse model of Barth syndrome
Barth syndrome is a rare metabolic disease caused by mutation of a gene called tafazzin or TAZ. It can cause life-threatening heart failure and also weakens the skeletal muscles, undercuts the immune response, and impairs overall growth. Because Barth syndrome is X-linked, it almost always occurs in boys. There is no cure or specific treatment. ... Read More about Gene therapy reverses heart failure in mouse model of Barth syndrome
Long-term hemophilia treatment could lie in patients’ own cells
Children (and adults) with hemophilia are slow to form blood clots, so are at constant risk for uncontrolled bleeding. Even when the skin isn’t broken, a fall or a simple toe stub can become a serious medical issue: internal bleeding cause permanent damage to muscles and joints. While regularly replacing the missing or malfunctioning clotting ... Read More about Long-term hemophilia treatment could lie in patients’ own cells