Gene therapy is the use of genes as treatment for a genetic disease by directing the expression of a therapeutic protein or restoring the expression of a missing protein. It involves the transfer of a therapeutic or working gene copy into specific cells of an individual to repair a faulty gene copy or to introduce a new gene whose function is to cure or to favorably modify the clinical course of a condition. This approach is different from traditional drug-based approaches, which may treat symptoms but not the underlying genetic problems. The scope of this approach is broad and promising for a wide number of diseases including inherited disorders, some types of cancer, and certain serious viral infections.
In a few years, genetic counselors will be in high demand so folks can make better decisions about their health. It can help manage a number of diseases by leveraging genes instead of drugs or surgery.
Although gene therapy shows promise, there are still risks involved, including unwanted immune system reactions or the risk of the wrong cells being targeted.
The nanoparticle delivers gene therapies to the right place in the body to fight disease. Dahlman is laser focused on ensuring the nanoparticles know what paths to take to reach the correct organ to start the healing process.
In my lab, we design different nanoparticles to deliver the genetically-engineered drugs to the correct location. During the course of identifying effective nanoparticles for drug delivery, thousands of nanoparticles must be tested, which presents scalability issues. But ethically, researchers cannot inject thousands of mice for an experiment of this magnitude.
So Dahlman developed a testing system that leverages DNA barcodes a stand-in for the actual drugs to label each nanoparticle. Once those are injected, researchers can see where the barcodes went in the mouse.
For example, if a significant number of barcodes numbered 30 all went to the heart, Dahlman can deduce that the nanoparticle represented by barcode 30 is best suited for that organ.
Second, we can now study the biology of drug delivery, understanding which genes affect how well a drug will work. And third, we can apply big data and artificial intelligence to drug delivery for the first time.
With thousands of nanoparticles being tested at once, we can mine giant data sets for bioinformatics.
Labs across the country can leverage FIND to accelerate their studies. If the technology is used by more labs, Dahlman believes it will increase the rate that gene therapies are developed, advancing the entire field. Non-liver gene therapy delivery is one of the biggest challenges today that Dahlman hopes to contribute to with his work.
The liver has been easier to target with gene therapy because of its filtration system; larger blood vessels let the nanoparticles pass more easily into the organ. Diseases such as hepatitis and cirrhosis have responded well to gene therapies.
The liver is responding extremely well to these therapies, and we are healing livers and curing people. The next frontier will be organs other than the liver, like the heart and brain with tighter blood vessel systems. That was one thing that really attracted me to Tech — the young faculty seemed truly happy here.
He never wants to be in a rut, and he wants to have the courage to pursue interesting and risky science even if lower risk work is safer. In his lab, his goal is to produce great students. In 20 years, he wants to see his students as leaders in the field.Perspective The Next Phase of Human Gene-Therapy Oversight Original Article Effect of Genetic Diagnosis on Patients with Previously Undiagnosed Disease Editorial The Coming of Age of Drug.
Erectile dysfunction adversely affects up to 20% of all men and is the most commonly treated sexual disorder. The public health implications of this condition are significant and represent a challenge for our healthcare system.
At the near-four month mark of , investors remain eager to find new opportunities—and various industries in the life science sector are certainly piquing the interest of savvy investors. BioMarin’s Gene Therapy Facility The Next Frontier: Investigational Gene Therapy for Hemophilia A In , Dr.
Barrie Carter, VP of Vector Biology at the science to the clinic for patients with hemophilia A, both as a treating physician and contact BioMarin Medical Information at . The next frontier of precision medicine: Parkinson's disease. Penn Medicine News | October 02, makeup can be analyzed to improve a clinician’s ability to diagnose Parkinson’s and select the right type of therapy for each individual.
mutations in the GBA gene occur in 5% to 10% of Parkinson’s patients, and treatments targeting this. Elsewhere, breakthroughs in medical science are making highly personalized medicine a reality. Personalized medicine is about delivering the right drugs and treatments to the right patients.