Gene silencing and gene editing: Page 2

U.K. regulators approve 'world-first' sickle-cell gene therapy
By Matt Limb
U.K. medical regulators have approved a “world-first” gene therapy that aims to cure two blood disorders: sickle cell disease and transfusion-dependent β-thalassemia. They are the first regulatory body to approve the treatment.
November 20, 2023
Sickle Cell Anemia2 Social
U.K. trial approved for Hunter syndrome gene therapy
By Matt Limb
U.K. regulators have approved the “first ever” trial of a revolutionary gene therapy for young children diagnosed with Hunter syndrome, a rare lysosomal storage disorder.
November 9, 2023
Gene Therapy Dna Scientist Social
CRISPR Therapeutics to present data from gene therapy programs at AHA Scientific Sessions
By LabPulse.com staff writers
Biopharmaceutical company CRISPR Therapeutics will present preclinical data from its investigational gene therapy treatment programs for cardiovascular disease at the AHA Scientific Sessions 2023 in Philadelphia on November 11.
November 6, 2023
Crispr Cas9 Gene Editing Social
Vertex Pharmaceuticals, CRISPR Therapeutics gene-editing therapy to undergo FDA advisory committee review
By LabPulse.com staff writers
An FDA advisory committee is scheduled to meet on October 31 to discuss and make recommendations on Vertex Pharmaceuticals' and CRISPR Therapeutics’ CRISPR-Cas9 gene-editing-based therapy, exagamglogene autotemcel.
October 30, 2023
Fda Sign Dc Social
Software reduces DANGER of CRISPR-Cas9 by shedding light on off-target edits
By Nick Paul Taylor
A new software can improve the safety and utility of CRISPR-Cas9 gene editing by detecting off-target changes and eliminating the need for reference genomes, according to a paper published in Bioinformatics Advances.
October 27, 2023
Crispr Cas9 Gene Editing Social
CRISPR-Cas3 technique restores dystrophin function in iPSCs from DMD patients
By Maryam Payne
Researchers have developed a dual CRISPR-Cas3 gene editing technique to restore dystrophin function in induced pluripotent stem cells (iPSCs) from patients with Duchenne muscular dystrophy (DMD).
August 24, 2023
This photo shows green dystrophin protein restored by dual-Cas3 in skeletal muscle derived from DMD patient iPSCs.
Messenger RNA shows preclinical promise as in vivo gene editing treatment for sickle cell
By Nick Paul Taylor
The delivery of the editing system resulted in the near-complete correction of hematopoietic sickle cells. A preclinical test linked the approach to a 91.7% increase in functional hemoglobin and a significant fall in the number of sickled cells.
August 14, 2023
Sickle Cell Anemia2 Social
Netherlands researchers combine CRISPR, bioluminescence in proof-of-concept infectious disease test
By Elissa Wolfson
Glow-in-the-dark proteins, developed from the luciferase found in bioluminescent creatures, may make viral disease diagnosis quicker and easier, according to the researchers.
March 15, 2023
Crispr Cas9 Gene Editing Social
Sherlock Biosciences nabs additional patent rights to Cas12 enzyme for CRISPR diagnostics
By LabPulse.com staff writers
Sherlock Biosciences has exclusive U.S. rights to the patent from Shanghai-based Tolo Biotech, as well as to additional intellectual property for Cas12 and Cas13 obtained from the Broad Institute.
February 21, 2023
Ms Crisp Rimage Social
Sherlock Biosciences acquires Sense Biodetection to drive CRISPR-based test adoption
By Leo O'Connor
The acquisition enables Sherlock to combine its CRISPR, synthetic biology, and artificial intelligence technologies with Sense's instrument-free diagnostic hardware and rapid molecular amplification chemistries.
February 1, 2023
Crispr Cas9 Gene Editing Social
AI tool makes speedy gene-editing possible, offers alternative to CRISPR
By Elissa Wolfson
An artificial intelligence program may enable the first simple production of customizable proteins called zinc fingers to treat diseases by turning genes on and off.
January 26, 2023
Crispr Cas9 Gene Editing Social
Discovery of destructive CRISPR enzyme opens up diagnostic, therapeutic applications for molecular scissors
By Nick Paul Taylor
In a pair of Nature papers, researchers describe the structure and function of Cas12a2, which binds and cuts target DNA to shut off a specific gene so that the enzymes can be used as molecular scissors.
January 4, 2023
Crispr Cas9 Gene Editing Social
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