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Published on October 29, 2024
U.S. researchers have shone further light into the precise way that CRISPR/Cas9 gene editing actually works and circumvents unintended off-target effects, which could lead to more accurate cellular therapies. The preclinical research, published in the journal Cell Chemical Biology, outlines several steps that are necessary for the Cas9 complex to…
Published on July 11, 2024
Targeted editing of a micro(mi)RNA mutation could help preserve the hearing of some patients genetically predisposed to deafness, a pre-clinical study indicates. The findings in mice suggest that CRISPR-Cas 9 gene editing of a mutation in miRNA-96 (Mir96) could be a potential strategy for preserving auditory function in humans. So…
Published on February 8, 2024
Researchers have used a gene editing technique to treat a rare immune disorder that could help overcome concerns over the use of gene therapy in these patients. The treatment targets deficiencies in recombination activating genes (RAGs), which can affect the production of lymphocytes and cause severe immune disorders. Specifically, it…
Published on January 3, 2024
Gene editing firm Tome Biosciences has acquired startup Replace Therapeutics less than a month after Tome emerged from stealth with $213M in funding. This development adds fuel to an already hot field. In early December, Vertex and CRISPR Therapeutics’ Casgevy (exagamglogene autotemcel [exa-cel]) for sickle cell became the first ever…
Published on July 28, 2023
An RNA-based gene editing tool that alters red blood cells inside the body could provide a simpler and less toxic route to treating conditions such as sickle cell anemia and beta thalassemia, researchers report. The discovery, outlined in Science, offers an alternative to current gene therapy for these blood disorders,…
Published on March 30, 2023
A newly created nanoparticle that carries a messenger RNA (mRNA) payload that can be administered to the lungs may offer a method to provide an inhalable treatment for cystic fibrosis and other lung diseases. Designed by engineers at MIT and the University of Massachusetts Medical School and applied in a…
Published on January 13, 2023
CRISPR gene editing could protect cardiac tissue from damage caused by a stroke or heart attack, according to research published in the journal Science. Currently, CRISPR-Cas9 gene editing is typically used to correct specific genetic mutations before the onset of disease, with treatment usually offered to a limited group of…
Published on December 16, 2022
Research led by the University of Verona and the University of Harvard shows the importance of taking human genetic diversity into account when designing gene editing therapies for conditions such as sickle cell disease. A number of different CRISPR genome editing techniques are currently being developed to treat conditions such…
Published on November 16, 2022
Ionis Pharmaceutical and Metagenomi have entered a collaboration to pursue investigational drugs for up to four validated and novel genetic targets. Ionis is an antisense/RNA-targeting pioneer while Metagnomi specializes in gene editing. Ionis has the right to add four more targets upon achievement of pre-determined development milestones. Under the terms…
Published on October 6, 2022
Gene editing technology that deploys a peptide nucleic acid (PNA) within a biocompatible polymer nanoparticle can correct a common mutation that causes cystic fibrosis (CF), a study in mice has revealed. A single injection partially restored function of the F508del mutation in the CF transmembrane conductance regulator (CFTR) protein, which is…
Published on September 7, 2022
Orbital Therapeutics, a Cambridge, Massachusetts-based RNA medicines startup, has launched with a view to developing new RNA therapeutics not including those based on RNA interference (i). The initial funding for Orbital is led by Arch Venture Partners and a16z Bio + Health and Newpath Partners have also contributed funds. The…
Published on April 25, 2022
Researchers based at the Center for Genome Engineering in the Republic of Korea have developed a new gene editing tool that can correct a single mutation in mitochondria, which the researchers hope will help develop treatments for serious human diseases caused by such mutations. Mitochondria are the so-called “powerhouses” of…
Published on April 11, 2022
Genome editing company Editas Medicine announced today the first administration of its therapy candidate EDIT-101 for the treatment of Leber congenital amaurosis 10 (LCA10), a CEP290-related retinal degenerative disorder. The BRILLIANCE clinical trial, designed to test the safety of EDIT-101, marks the first-ever delivery of an experimental CRISPR gene editing…
Published on February 8, 2022
Defective mitochondria could be repaired using gene-editing, according to work from scientists at the University of Cambridge. They have modified the mitochondrial genome in live mice, paving the way for new treatments for incurable mitochondrial disorders. Their work is published today in Nature Communications. Pedro Silva-Pinheiro, a postdoctoral researcher and first…
Published on January 26, 2022
Metagenomi completed an oversubscribed $175 million Series B financing this week, which it says will enable it to advance its lead in vivo and ex vivo gene editing therapy programs into the clinic. The developer of CRISPR-based gene editing systems for developing cell and gene therapies also said the financing…