Abstract In myotonic dystrophy type 1 (DM1), deregulated alternative splicing of the muscle chloride channel Clcn1 causes myotonia, a delayed relaxation of muscles due to
Summary Microhomology-mediated end joining (MMEJ) is an intrinsically mutagenic pathway of DNA double-strand break (DSB) repair essential for proliferation of homologous recombination (HR)-deficient tumors. Although targeting MMEJ
Abstract Class 2 CRISPR effectors Cas9 and Cas12 may have evolved from nucleases in IS200/IS605 transposons. IscB is about 2/5 the size of Cas9 but
Abstract Genetic intervention is increasingly being explored as a therapeutic option for debilitating disorders of the central nervous system. The safety and efficacy of gene
Hitching a ride with a retroelement Retroviruses and retroelements have inserted their genetic code into mammalian genomes throughout evolution. Although many of these integrated virus-like
AbstractWith the development of CRISPR/Cas9-mediated gene editing technologies, correction of diseasecausing mutations has become possible. However, current gene correction strategies preclude mutationrepair in post-mitotic cells
Abstract RNAi therapy has undergone two stages of development, direct injection of synthetic siRNAs and delivery with artificial vehicles or conjugated ligands; both have not
Abstract Leber congenital amaurosis due to CEP290 ciliopathy is being explored by treatment with the antisense oligonucleotide (AON) sepofarsen. One patient who was part of a larger
Abstract Genetic variants account for approximately half the cases of congenital and early‐onset deafness. Methods and technologies for viral delivery of genes into the inner
Unexpected transfer Gene therapy using adeno-associated viral (AAV) vectors is a promising strategy for monogenic blinding diseases. Leber hereditary optic neuropathy (LHON) is caused by
Madison researchers have developed a safer and more efficient way to deliver a promising new method for treating cancer and liver disorders and for vaccination
Highlights COPD is the third leading cause of death globally, and there is an urgent unmet medical need to develop novel therapeutics specifically for COPD.
In late 2017, scientists first began attempting to edit the genes of adults to treat rare genetic disorders. Preliminary results from a clinical trial, shared
Two gene injections reprogram Müller glia into rod photoreceptors in the retinas of blind mice and partially restore the animals’ vision, researchers report today (August
Researchers have successfully treated animals with a mouse version of type 1 diabetes by inducing pancreatic cells that don’t normally produce insulin to manufacture the
In a first, a man has received a therapy aimed at editing the genes inside his body. The Associated Press reports that 44-year-old Brian Madeux,
Early phase Northwestern Medicine research published in the journal Proceedings of the National Academy of Sciences has demonstrated a potential new therapeutic strategy for treating
Cold Spring Harbor Laboratory molecular geneticist Adrian Krainer was at a National Institutes of Health workshop in 1999 when he first learned about the crippling
Preserving the distinction between research purposes and clinical applications, the Human Fertilisation and Embryology Authority (HFEA), a U.K. regulatory body, has approved the use of
The hottest technique in gene editing, called CRISPR, could soon be used to study human embryos. On 14 January, a regulatory committee in the United
Adenovectors are hot again. Two decades after the initial flurry of excitement about gene-based medicine, researchers and the media are once again enthusiastic about the
If a Cas9 nuclease variant could be engineered that was less grabby, it might loosen its grip on DNA sequences throughout the genome except those
Highlights Small-molecule inhibition of RAD52 activity in vitro and in vivo Small-molecule dimerization of RAD52 undecamer rings Small-molecule disruption of RAD52 ring superstructures Small-molecule inhibition of RAD52
Chinese stem-cell scientists have welcomed long-awaited measures that, state media claim, will rein in rogue use of stem cells in clinics while allowing research. The
Scientists have used two methods to generate patient-specific pluripotent stem cells with normal mitochondria for people with defects in these organelles, according to a study
Created by ePubSystems. Contact Us for similar site for your university or institute.