Cancer & Disease Research
Potential of gene editing for clinical use highlighted at ASGCT 2021
From base editing to in vivo CRISPR therapeutics and CRISPR-modified bacteriophages, scientists discussed innovations in preclinical research that have allowed them to advance these unique products to the clinic during a scientific symposium at the 2021 American Society of Gene & Cell Therapy (ASGCT) virtual meeting. Read More
Nobel Prize winner Doudna shares perspectives on future of CRISPR gene editing
Small science can translate into big discoveries, according to Nobel Prize laureate Jennifer Doudna, PhD. She discussed how her curiosity in understanding CRISPR led to the gene editing revolution in a talk at the 2021 American Society of Gene & Cell Therapy virtual meeting. Read More
Development 'cookbooks' could speed up gene therapy
Great strides have been made in the development of gene editing tools in the past decade, but translating the technology into real therapies to treat patients has taken longer than expected. How to accelerate this process was the subject of a panel discussion on January 13 at the Biotech Showcase virtual event. Read More
Is 'bespoke' therapy the future of genomic medicine?
Gene therapies hold extremely exciting promise for meeting the unmet needs of many individuals with genetic diseases, as discussed in a session of the second annual American Society of Gene & Cell Therapy Policy Summit on September 24. Read More
Customized gene therapies successfully target rare eye diseases
Can gene therapies prove effective for the treatment of rare genetic diseases? One company may have found a solution that incorporates a high degree of customization, manufacturing expertise, and years' worth of knowledge. Read More
Genomic mutations can influence disease risk
The reason why some genetically predisposed individuals may or may not develop a disease is rooted in mutations throughout the genome, according to a new study published in Nature Communications on August 20. The researchers explained how this information can be used to improve disease risk estimations in the clinic. Read More
Next-generation sequencing reveals inherited mutations in young cancer patients
Researchers advocated for increased genomic surveillance in younger adult cancer patients with tumors that typically strike later in life, in a study presented at the American Association for Cancer Research 2020 virtual meeting, held June 22 to 24. Read More
Spell-check gene editing corrects hearing loss in mice
Using a new genetic engineering technique called base editing, researchers restored genetic hearing loss in mice with a recessive point mutation. The research was published by a group from Boston Children's Hospital, the Broad Institute of the Massachusetts Institute of Technology, and Harvard University in Science Translational Medicine on June 3. Read More
Forward-oriented gene therapy improves treatment for sickle cell disease
Researchers at the National Institutes of Health (NIH) have developed a new and improved viral vector that is up to 10 times more efficient at incorporating corrective genes into bone marrow stem cells than conventional treatments. The work was published in Nature Communications on October 2, and was supported by the National Heart Lung, and Blood Institute (NHLBI) and the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) at the NIH. Read More
A novel approach to using CRISPR: curing cystic fibrosis
Researchers are finding innovative ways to utilize CRISPR technology to permanently cure diseases, in this case, cystic fibrosis. A collaboration between the University of Trento in Italy and KU Leuven in Belgium, funded by Fondazione ricerca fibrosi cistica led to the publication of a new study in Nature Communications on August 7, 2019. Read More
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