Archive for gene therapy
Optimized CRISPR/Cas9 gene editing averts hearing loss in ‘Beethoven’ mice
Using a novel gene-editing approach, scientists at Boston Children’s Hospital and Harvard Medical School have salvaged hearing in a mouse model of hereditary deafness, with no apparent off-target effects. The system successfully identified a single misspelled “letter” in the defective copy of a gene required for hearing, disabled this aberrant copy, and spared the healthy ... Read More about Optimized CRISPR/Cas9 gene editing averts hearing loss in ‘Beethoven’ mice
Tagged: gene therapy, genetics and genomics, hearing, ophthalmology
Our SCID gene therapy journey
In just 18 months, our son Paul Gallagher has been through more than what most people go through in a lifetime. He was born on Nov. 10, 2017, with a tenacious spirit, yet without an immune system — meaning that the simplest infection was difficult to fight and a common cold was life threatening. His ... Read More about Our SCID gene therapy journey
Tagged: gene therapy, scid
Cole: Having a ball after CAR T-cell therapy
After undergoing a promising new treatment for acute lymphoblastic leukemia (ALL), Cole Malone is back to doing what he loves: playing on a flag football team with his twin brother, Michael. Cole and Michael, 14, already know plenty about teamwork. Michael served as a perfect-match donor when Cole had a stem cell transplant at Dana-Farber/Boston Children’s Cancer and Blood ... Read More about Cole: Having a ball after CAR T-cell therapy
The space between heartache and happiness: Two sons with adrenoleukodystrophy
When Paul and Liliana Rojas talk about their life, they describe it in one of two ways — the way it was before their sons, 10-year-old Brandon and 7-year-old Brian, were diagnosed with ALD, and the way it is after. Their story is one of heartbreak — but also hope, in the form of a ... Read More about The space between heartache and happiness: Two sons with adrenoleukodystrophy
Tagged: adrenoleukodystrophy, clinical trials, gene therapy
Manny: Hoping new research helps others with sickle cell disease
Emmanuel “Manny” Johnson, Jr., shares many loves with his little brother, Aiden — from basketball to video games. One thing he wishes they did not share is sickle cell disease (SCD), so Manny is playing a role in a new effort to improve treatment for patients like 7-year-old Aiden, himself and others living with the inherited blood disorder. ... Read More about Manny: Hoping new research helps others with sickle cell disease
Tagged: gene therapy, sickle cell disease
Reviving fetal hemoglobin in sickle cell disease: First patient is symptom-free
Manny Johnson of Boston, 21, previously required monthly blood transfusions to keep his severe sickle cell disease under control. After receiving a new gene therapy treatment, he’s been symptom-free for six months. Researchers at the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center reported Manny’s case Saturday at the American Society of Hematology meeting in San Diego. Manny ... Read More about Reviving fetal hemoglobin in sickle cell disease: First patient is symptom-free
Tagged: blood, clinical trials, gene therapy, sickle cell disease
Sickle cell gene therapy to boost fetal hemoglobin: A 70-year timeline of discovery
Boston Children’s Hospital is now enrolling patients age 3 to 35 in a clinical trial of gene therapy for sickle cell disease. Based on technology developed in its own labs, it differs from other gene therapy approaches by having a two-pronged action. It represses production of the mutated beta hemoglobin that causes red blood cells to ... Read More about Sickle cell gene therapy to boost fetal hemoglobin: A 70-year timeline of discovery
Tagged: blood, gene therapy, hematology, sickle cell disease
Gene therapy halts progression of cerebral adrenoleukodystrophy in clinical trial
Adrenoleukodystrophy — depicted in the 1992 movie “Lorenzo’s Oil” — is a genetic disease that most severely affects boys. Caused by a defective gene on the X chromosome, it triggers a build-up of fatty acids that damage the protective myelin sheaths of the brain’s neurons, leading to cognitive and motor impairment. The most devastating form of the ... Read More about Gene therapy halts progression of cerebral adrenoleukodystrophy in clinical trial
Tagged: adrenoleukodystrophy, gene therapy
Landmark moment for science as the FDA approves a gene therapy for the first time
Today, the Food and Drug Administration approved a gene therapy known as CAR T-cell therapy that genetically modifies a patient’s own cells to help them combat pediatric acute lymphoblastic leukemia (ALL), the most common childhood cancer. It is the first gene therapy to be approved by the FDA. “This represents the progression of the field of gene ... Read More about Landmark moment for science as the FDA approves a gene therapy for the first time
Tagged: acute lymphoblastic leukemia, cancer, gene therapy
BCL11A-based gene therapy for sickle cell disease passes key preclinical test
Research going back to the 1980s has shown that sickle cell disease is milder in people whose red blood cells carry a fetal form of hemoglobin. The healthy fetal hemoglobin compensates for the mutated “adult” hemoglobin that makes red blood cells stiffen and assume the classic “sickle” shape. Normally, fetal hemoglobin production tails off after birth, shut ... Read More about BCL11A-based gene therapy for sickle cell disease passes key preclinical test
Tagged: blood, gene therapy, sickle cell disease