Thanks to gene editing, we’ve seen much progress in hard-to-treat conditions. Sufferers of both muscular dystrophy and leukemia are experiencing a new variety of treatment options. Now, thanks to CRISPR, a renowned gene editing tool, researchers have increased HIV resistance in animals.
A minor proportion of people harbor a homozygous mutation in CCR5—a gene that encodes a receptor found on immune cells—that thwarts HIV’s attempts to get inside the cells. In an attempt to mimic this natural resistance, researchers mutated CCR5 in human fetal liver hematopoietic stem/progenitor cells (HSPCs) and showed that the cells could block HIV infection after transplantation into mice.
Don’t let the medical jargon fool you — while the procedure may be complicated, the concept itself is a lot simpler. By replicating a naturally occurring genetic mutation, T-cells become more resistant to viruses. But results were slow, and researchers were patient, to say the least.
“The long-term reconstitution and secondary transplantation were time-consuming. It took us more than one-year monitoring of the mice to confirm the gene editing is robust in long-term HSCs,”
The study may not have been the first to incorporate gene editing, but it is the first to use CRISPR. We may not have engineered a complete cure (after all, we’ve only targeted mice), but finding one wouldn’t seem too improbable.
Moral of the story? Take risks. Sponsor a child genius. Our future depends on them.
Treatments for the seeing-impaired are not always easy to come by. That’s why we make do with technology like talking cameras that allow the blind to “see.” However, a new study shows that mimicking fish eyes could potentially cure blindness.
Researchers at the University of Washington in Seattle reported that they have hacked the cells of a mouse retina to act like those of a fish—not only growing new neurons, but also wiring those neurons up to other neurons that send signals to the brain.
While surgery can treat cataracts, retina damage is incurable — but not for zebrafish. Their eyes regenerate indefinitely, assisted by a cell called Müller glia. The cell acts as a “stand in” for lost neurons. Humans also carry the cell but due to differences in DNA, cannot access this reprogrammable characteristic.
[UW Researchers found] Trichostatin-A (TSA), a hormonal treatment for breast cancer, that also happens to open up the regeneration DNA sequence. In an injured retina, these Müller glia cells treated with TSA transformed into two types of neurons, bipolar and amacrine cells, that are part of the retina’s internal wiring.
Scientists have yet to produce photoreceptor neurons, but with the way things are going, creating them is very possible.
Alongside scientists, mice have played a huge role in health-related breakthroughs. Since previously advancing diabetes research, mice have now led us to a potential cure for paralysis. In particular, gene editing has eliminated muscular dystrophy in mice.
The mice in the study have a rare and severe form of congenital muscular dystrophy known as MDC1A.
The illness is caused by a splice site mutation: a genetic error makes cellular messengers misread a critical section of DNA, like the scratch that makes a record skip.
Researchers in Cohn’s lab used CRISPR to cut out the scratch. Natural cell repair mechanisms stitched the remaining strands of DNA back together, allowing the whole genetic sequence to be read normally.
Follow ups proved that the participating mice were healed completely. The method is also simple, unlike other processes that require the engineering of an entirely new genome. However, as with all animal testing, researchers must carefully consider human trials.
For the first time it’s possible to think about — and this is still at the thinking stage, let’s be clear — the possibilities of gene correction in humans with these diseases,”
Considering that patients with muscular dystrophy rarely make it through their twenties, this could be the change they need. It may be a while, but gene editing holds much promise for the future.
Often the guinea pigs of science, mice have surely seen better days. However, in a recent study on treatment options for diabetes, the mice tested experienced no side effects. In fact, they may have led us to a cure.
The discovery, made at The University of Texas Health Science Center… increases the types of pancreatic cells that secrete insulin.
UT Health San Antonio researchers have a goal to reach human clinical trials in three years, but to do so they must first test the strategy in large-animal studies, which will cost an estimated $5 million.
Talk about cash for a cause! To achieve the cure, researchers used a therapy method called gene transferring.
A virus is used as a vector, or carrier, to introduce selected genes into the pancreas. These genes become incorporated and cause digestive enzymes and other cell types to make insulin.
The therapy regulates blood sugar in mice with extreme accuracy, which is something insulin hasn’t quite mastered. Gene transferring, however, replicates the characteristics of lost beta cells in diabetics.
So far, 2017 has been a milestone year for medical advancements. Alongside diabetes, treatments for ALS have also seen some improvements. So whether we’d like to think otherwise, a lot of medical breakthroughs wouldn’t be possible without our good old friend Mickey.