Archive for gene editing
Researchers identify genes that could affect children’s bone growth
Many children anticipate reaching the next number on a measuring stick as they chart their growth. But measurements can be frustrating or stressful for children who have skeletal disorders that prevent bones from growing. Key findings Researchers used genome-wide association studies and CRISPR gene editing to see if alterations to cartilage cells, which are known ... Read More about Researchers identify genes that could affect children’s bone growth
Sickle cell gene therapy and boosting fetal hemoglobin: A 75-year history
Ed. Note: This post updates an earlier post from 2018. In a landmark decision today, the Food and Drug Administration (FDA) approved two gene therapies for sickle cell disease. One of them, Casgevy, has deep scientific roots at Boston Children’s Hospital — and is also the first therapy using CRISPR gene editing to gain FDA ... Read More about Sickle cell gene therapy and boosting fetal hemoglobin: A 75-year history
Making immunotherapy safe for AML
Acute myeloid leukemia (AML), the second most common leukemia in children, is hard to treat and has a five-year survival rate of just 65 to 70 percent, according to the American Cancer Society. While immunotherapies like monoclonal antibodies or CAR T-cell therapy are effective for certain blood cancers, they have not been possible in AML ... Read More about Making immunotherapy safe for AML
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 about A potential danger of CRISPR gene editing — and why base editing may be safer
Looking for cancer’s Achilles heel: The Pediatric Cancer Dependency Map
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 ... Read More about Looking for cancer’s Achilles heel: The Pediatric Cancer Dependency Map
Avoiding a lifetime of injections: Can gene editing cure severe congenital neutropenia?
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 ... Read More about Avoiding a lifetime of injections: Can gene editing cure severe congenital neutropenia?
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 first for CRISPR: Cutting genes in blood stem cells
CRISPR — a gene editing technology that lets researchers make precise mutations, deletions and even replacements in genomic DNA — is all the rage among genomic researchers right now. First discovered as a kind of genomic immune memory in bacteria, labs around the world are trying to leverage the technology for diseases ranging from malaria ... Read More about A first for CRISPR: Cutting genes in blood stem cells
Genome editing: A CRISPR way to correct disease
Technology sometimes unfolds at a slow, measured pace and sometimes at lightning speed. Right now, we are witnessing what is arguably one of the fastest moving fields in biomedical science: a form of genome editing aptly known as CRISPR. CRISPR allows researchers to make very precise—some would say crisp—changes to the genomes of human cells ... Read More about Genome editing: A CRISPR way to correct disease