UAB Researchers Use CRISPR Technology to Target Childhood Syndrome
In the last couple of years there’s been a bonanza in life sciences around something called CRISPR. This is a process that bacteria do naturally as a defense mechanism, but scientists have figured out how to use it to snip out specific genes from DNA and replace them. In other words, it makes cheaper and more precise genetic editing possible.
“This kind of CRISPR technology that many people are practicing now is going to change the whole field of medicine,” says Tim Townes, director of the UAB Stem Cell Institute.
Townes led a team that published a paper Thursday in Cell Reports on their use of CRISPR to develop a potential new cure for a troubling condition in children. It’s called severe combined immunodeficiency or SCID. Children born with SCID have no immune response so they can’t fight invading viruses or bacteria. It’s more popularly know as the “bubble boy” syndrome. The only current treatment is a bone marrow transplant. Otherwise SCID is fatal.
Townes received skin cells from a patient who was successfully cured through a bone marrow transplant. His team then changed those cells to stem cells and used the CRISPR technique to change the one DNA base pair that causes SCID. The idea is that those cells would then be converted to blood stem cells and transplanted back into the patient, curing him or her.
“So they would be getting their own cells. But those cells would be corrected,” said Townes.
The Food and Drug Administration hasn’t allowed any studies on people using CRISPR yet, but Townes expects UAB to start clinical trials on the SCID therapy within two years.
While CRISPR has the potential to revolutionize biology, from curing genetic diseases to creating mosquitos that can’t carry malaria, it comes with weighty ethical concerns. Scientists and bioethicists are already warning about unintended consequences from changed genes, particularly if they are passed on to children. Also, CRISPR could theoretically be used to by parents to create “designer babies,” genetically engineered for desirable traits.
Scientists in China prompted an uproar earlier this year when they announced they used CRISPR to alter genes in human embryos.
Townes said they check carefully they only change the base pair targeted. He also says they’re only interested in curing disease.
“Any technology can be used for good or not so good purposes,” said Townes. “We want to develop new technology and it’s up to society as a whole to apply them in the correct way.”
Extra: Radiolab explains CRISPR
Workers at Alabama’s Mercedes plants vote against joining a union
The workers voted 56% against the union, according to tallies released by the National Labor Relations Board, which ran the election.
Taylor Hunnicutt is championing her home state with debut album ‘Alabama Sound’
Alabama offers a diverse symphony of swampy rock, swaggering blues, murky country, and bone-rattling soul. It’s a sound singer-songwriter Taylor Hunnicutt recently reimagined on her debut album, Alabama Sound, which is out now.
UAB building among Alabama’s first to feature ‘bird safe’ glass
Advocates hope the new glass will prevent window collisions, a leading causes of bird mortality.
Researchers look into community health impact of wood pellet production in rural Mississippi
Brown University and Tougaloo College students are testing for potential air and noise pollution near the Drax wood pellet plant in Gloster, Mississippi.
Alabama coal company sued for a home explosion is delinquent on dozens of penalties
Crimson Oak Grove Resources has been cited for 204 safety violations since the March 8 blast, many involving “significant and substantial” safety violations.
The United Auto Workers faces a key test in the South with upcoming vote at Alabama Mercedes plant
The United Auto Workers is aiming for a key victory at Mercedes-Benz in Alabama. More than 5,000 workers at the facility in Vance and nearby battery plant will vote this week on whether to join the UAW.
