Partnership to develop gene editing therapies for neurological, muscular diseases
Regeneron Pharmaceuticals Inc. and Intellia Therapeutics Inc. have announced an expanded research collaboration aimed at developing further CRISPR-based gene-editing therapies focused on neurological and muscular diseases. According to an Intellia press release, the expanded partnership aims to combine Regeneron’s proprietary adeno-associated viral vectors and antibody-targeted delivery systems with Intellia’s proprietary Nme2 CRISPR/Cas9 systems tailored to […]
Small and precise CRISPR-Cas13bt3 system can be used to shred viruses
There are different types of CRISPR system, and the one on which researchers have focused to describe the three-dimensional structure in detail is called CRISPR-Cas13bt3. What’s unique about it is that it’s very small. Usually, these types of molecules contain around 1,200 amino acids, whereas this one only contains around 700. Smaller size is a […]
New CAR T cell therapy approach minimizes severe side effects
Six CAR T cell therapies have received FDA approval, and several more are in the pipeline. However, these therapies carry serious and potentially fatal side effects, namely cytokine release syndrome (CRS) and neurotoxicity. These drawbacks manifest themselves in a range of symptoms, from high fever and vomiting to multivisceral failure and patient death, posing significant […]
NICER: A safer alternative to CRISPR/Cas9 for gene editing
CRISPR/Cas9 method can lead to unintended DNA mutations that can have negative effects. Recently, Japanese researchers have developed a new gene-editing technique that is as effective as CRISPR/Cas9, yet significantly reduces these unintended mutations. In a new study published in Nature Communications , researchers led by Osaka University have introduced a new technique called NICER, […]
Universal approach could potentially expand CAR T cell therapy to all blood cancers
Until now, researchers have lacked the tools to create a targeted cell therapy approach that could work on all the different forms of blood and bone marrow cancers. A new solution could solve a major problem in immunotherapy, namely the inability to target surface markers present on both cancerous and healthy cells. In the study, […]
Ovarian cancer patients enrolled in first-in-human trial of CAR T cell therapy
Researchers from one of the largest cancer research and treatment organizations in the USA, have published preclinical research in Nature Communications demonstrating that a CAR T cell therapy targeting TAG72, a target found on the surface of ovarian cancer cells, eradicates cancer cells in mouse models. The researchers also found that by adding the cytokine […]
Novel gene editing cell therapy shows curative potential in sickle cell disease
An experimental gene-editing cell therapy for sickle cell disease has shown encouraging initial results in a multicenter Phase 1 and Phase 2 clinical trial. The clinical trial is the first in which CRISPR-Cas12 is used to edit human cells. Cas12 has been shown in preclinical research to be more effective for gene editing as it […]
Novel gene editing cell therapy shows curative potential in sickle cell disease
An experimental gene-editing cell therapy for sickle cell disease has shown encouraging initial results in a multicenter Phase 1 and Phase 2 clinical trial. The clinical trial is the first in which CRISPR-Cas12 is used to edit human cells. Cas12 has been shown in preclinical research to be more effective for gene editing as it […]
New study warns of dangers of gene editing in human embryos
A new study highlights the limits of gene editing in human embryos. Researchers have used CRISPR-Cas9 in an attempt to correct genetic errors in human embryos. However, embryonic cells have difficulty repairing broken DNA strands, leading to further mutations. In one study, researchers fertilized donated eggs with donated sperm using intracytoplasmic sperm injection (ICSI) to […]
New study warns of dangers of gene editing in human embryos
A new study highlights the limits of gene editing in human embryos. Researchers have used CRISPR-Cas9 in an attempt to correct genetic errors in human embryos. However, embryonic cells have difficulty repairing broken DNA strands, leading to further mutations. In one study, researchers fertilized donated eggs with donated sperm using intracytoplasmic sperm injection (ICSI) to […]
