Archive for gene therapy
Good early results with gene therapy for rare immune deficiency
Brenden Whittaker, a college student in Ohio, has been caught off guard by his good health. Since he was young, a rare immune deficiency known as chronic granulomatous disease (CGD) had left him vulnerable to life-threatening infections. He was used to going in and out of the hospital, and then hooking up to an IV ... Read More about Good early results with gene therapy for rare immune deficiency
Bone marrow-on-a-chip provides new research directions for Shwachman-Diamond syndrome
A new research tool that mimics the behavior of diseased bone marrow provides a new strategy for understanding the bone marrow disease, Shwachman-Diamond syndrome (SDS), and hopefully, developing new treatments. With SDS, bone marrow fails to produce blood cells normally, leading to bone marrow failure and an increased risk of leukemia. In a research paper ... Read More about Bone marrow-on-a-chip provides new research directions for Shwachman-Diamond syndrome
Gene therapy to boost fetal hemoglobin continues to do well in sickle cell trial
A pilot gene therapy treatment for sickle cell disease, restoring patients’ ability to make fetal hemoglobin, has produced good results in the first three patients to receive it. Investigators at Boston Children’s Hospital reported the findings of their ongoing clinical trial this week at the American Society of Hematology (ASH) annual meeting. The three adult ... Read More about Gene therapy to boost fetal hemoglobin continues to do well in sickle cell trial
Sofie’s story: A new gene therapy treatment for SMA
Sofie Petrovická sits up in the exam room, smiling and laughing with her physical therapist, Elizabeth Maczek. Though she’s just over a year old, sitting on her own is no minor feat for Sofie — in fact, it’s nothing short of a miracle. Sofie was just 3 months old when she was diagnosed with spinal ... Read More about Sofie’s story: A new gene therapy treatment for SMA
Novel CRISPR system could halt growth of triple-negative breast cancer
Triple-negative breast cancer (TNBC), lacking estrogen, progesterone and HER2 receptors, has the highest mortality rate of all breast cancers. It more frequently strikes women under age 50, African American women, and women carrying a BRCA1 gene mutation. The highly aggressive, frequently metastatic cancer is in urgent need of more effective targeted therapeutics. A new tumor-targeted ... Read More about Novel CRISPR system could halt growth of triple-negative breast cancer
Tissue models and a gene therapy for a deadly heart arrhythmia
Catecholaminergic polymorphic ventricular tachycardia (CPVT) is a leading cause of sudden death from cardiac arrest in children and young adults. Marked by racing, irregular heartbeats, the inherited arrhythmia is typically silent until a child suddenly collapses and faints, at an average age of 12. The trigger is an adrenaline surge, caused by exercise or emotional ... Read More about Tissue models and a gene therapy for a deadly heart arrhythmia
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
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
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