Genome editing to treat human retinal degeneration
New Rochelle, NY, January 19, 2021–Gene editing therapies, including CRISPR-Cas systems, offer the potential to correct mutations causing inherited retinal
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Biodistribution of AAV gene transfer vectors in nonhuman primate
New Rochelle, NY, January 15, 2021–The biodistribution of adeno-associated virus (AAV) gene transfer vectors can be measured in nonhuman primates
Read moreCHOP and Penn Researchers Identify Nanoparticles that Could Be Used in Therapeutic mRNA Delivery before Birth
Researchers at Children’s Hospital of Philadelphia and the School of Engineering and Applied Science at the University of Pennsylvania have identified ionizable lipid nanoparticles that could be used to deliver mRNA as part of fetal therapy. The proof-of-concept study, published today in Science Advances, engineered and screened a number of lipid nanoparticle formulations for targeting mouse fetal organs and has laid the groundwork for testing potential therapies to treat genetic diseases before birth.
Read moreResearchers identify nanoparticles that could deliver therapeutic mRNA before birth
Study demonstrates the efficacy and therapeutic potential of select ionizable lipid nanoparticles for delivering mRNA to treat genetic diseases before birth
Read moreA CNIO study links severe COVID-19 disease to short telomeres
The study was carried out with data from 89 patients admitted to the IFEMA Hospital
Read moreGene therapy strategy found effective in mouse model of hereditary disease TSC
Treatment reverses aspects of tuberous sclerosis complex, which is characterized by noncancerous tumors in multiple organs.
Read moreNew clues to prostate cancer
Why aggressive subtype can resist treatment
Read moreBrain cancer linked to tissue healing
Brain tumours might arise when tissue does not heal properly– a finding that opens up new ideas about how cancer develops and how to combat it
Read moreScientists at Tel Aviv University develop new gene therapy for deafness
Breakthrough may help in the treatment of children with hearing loss
Read moreSalk Assistant Professor Dmitry Lyumkis receives CAREER award from NSF
LA JOLLA–(December 22, 2020) Assistant Professor Dmitry Lyumkis has received a Faculty Early Career Development Program (CAREER) award from the
Read moreResearchers identify a rare genetic bone disorder through massive sequencing methods
They have used precision medicine to uncover and treat new skeletal disorders
Read moreGene therapy for placental insufficiency moves toward the clinic
New Rochelle, NY, December 15, 2020–A new study identified an adenovirus gene therapy vector carrying a VEGF isoform. It can
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