Patients’ own cells transformed into model for studying disease and developing potential treatment
Columbia University Medical Center (CUMC) researchers have created a way to develop personalized gene therapies for patients with retinitis pigmentosa (RP), a leading cause of vision loss. The approach, the first of its kind, takes advantage of induced pluripotent stem (iPS) cell technology to transform skin cells into retinal cells, which are then used as a patient-specific model for disease study and preclinical testing.
Using this approach, researchers led by Stephen H. Tsang, MD, PhD, showed that a form of RP caused by mutations to the gene MFRP (membrane frizzled-related protein) disrupts the protein that gives retinal cells their structural integrity. They also showed that the effects of these mutations can be reversed with gene therapy. The approach could potentially be used to create personalized therapies for other forms of RP, as well as other genetic diseases. The paper was published recently in the online edition of Molecular Therapy, the official journal of the American Society for Gene & Cell Therapy.
“The use of patient-specific cell lines for testing the efficacy of gene therapy to precisely correct a patient’s genetic deficiency provides yet another tool for advancing the field of personalized medicine,” said Dr. Tsang, the Laszlo Z. Bito Associate Professor of Ophthalmology and associate professor of pathology and cell biology.
The Latest on: Personalized Gene Therapy
via Google News
The Latest on: Personalized Gene Therapy
- New findings speed progress towards affordable gene therapyon November 20, 2020 at 9:41 am
In a promising advance for affordable, personalized medicine, researchers have used metal-organic frameworks to successfully deliver the genetic snipping tool CRISPR/Cas9 into human cancer cells.
- Approaching tumor diseases with targeted therapyon November 20, 2020 at 6:51 am
Personalized Immunotherapy for Tumor Diseases ... These developments include T-cell gene therapy and T-cell gene editing, transgenic T-cells for increasing affinity to tumor cells such as CAR ...
- Gene Therapy Market Share 2020, Trends, Size - Industry Report 2027on November 20, 2020 at 1:22 am
The need for tailored care and rising recognition and acceptance among patients of personalized medicines is likely to further activate the global gene therapy industry further in the future.
- AVROBIO Announces New Positive Clinical Data and Preclinical Data, as Well as Expanded Leading Lysosomal Disorder Gene Therapy Pipelineon November 18, 2020 at 3:05 am
- Boosting Capabilities in Gene and Cell Therapy Bioprocessingon November 17, 2020 at 1:31 pm
“There’s a gap around handling manufacturing data in cell and gene therapy manufacturing ... vast amount of data produced during personalized therapy manufacturing, where a different ...
- AVROBIO Announces New Positive Clinical Data and Preclinical Data, as Well as Expanded Leading Lysosomal Disorder Gene Therapy Pipelineon November 17, 2020 at 8:00 am
AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today an ...
- AVROBIO Announces New Positive Clinical Data and Preclinical Data, as Well as Expanded Leading Lysosomal Disorder Gene Therapy Pipelineon November 17, 2020 at 4:02 am
and the expected benefits and results of our implementation of the plato platform in our clinical trials and gene therapy programs, including the use of a personalized and ultra-precision busulfan ...
- Genomic Medicine: Slowly Moving To All At Onceon November 16, 2020 at 5:50 pm
Bryan Zeitler, Sangamo Therapeutics This precise and personalized approach to medicine ... are early validation efforts leading to gene therapy and allogeneic cell therapies from a slew of ...
- Monitoring the Off-Target Effects of Gene Editingon November 10, 2020 at 12:07 pm
KromaTiD’s dGH-in-Site™ service was recently launched with the aim of improving the safety of personalized medicines ... will also help cell and gene therapy manufacturers with their process ...
via Bing News