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Mutations responsible for common inherited heart condition corrected using CRISPR-Cas9 therapy

Using the CRISPR-Cas9 gene editing system, UT Southwestern researchers corrected mutations responsible for a common inherited heart condition called dilated cardiomyopathy (DCM) in human cells and a mouse model of the disease.

21 Dec 2022

CRISPR/Cas9-engineered avatar mouse model of rare disease paves the way for targeted therapy

An international research team led by Dr Ana Guadaño at the Alberto Sols Biomedical Research Institute (IIBM, a combined CSIC-UAM center) and involving the Complutense University of Madrid (UCM), used CRISPR gene editing techniques to incorporate into the mice a mutation of the MCT8 protein responsible for transporting thyroid hormones to the interior of the cell.

12 Dec 2022

GOSH delivers world-first base-edited cell therapy for a patient with relapsed T cell leukemia

A patient with relapsed T cell leukemia has been given base-edited T-cells in a world-first use of a base-edited cell therapy, in a 'bench-to-bedside' collaboration between UCL and Great Ormond Street Hospital for Children (GOSH).

12 Dec 2022

Salk researchers shed new light on specific mechanisms that activate oncogenes

Cancer, caused by abnormal overgrowth of cells, is the second-leading cause of death in the world. Researchers from the Salk Institute have zeroed in on specific mechanisms that activate oncogenes, which are altered genes that can cause normal cells to become cancer cells.

7 Dec 2022

Researchers uncover the host protease CAPN2 as a novel host factor that aids the infection of SARS-CoV-2

Researchers investigated the potential of the CAPN2 molecule as a therapeutic target against SARS-CoV-2.

5 Dec 2022

Researchers develop 3D organoid model to advance understanding of gastroesophageal junction cancer

Researchers at Johns Hopkins Medicine say they have created a laboratory-grown three-dimensional "organoid" model that is derived from human tissue and designed to advance understanding about how early stages of cancer develop at the gastroesophageal junction (GEJ) — the point where the digestive system's food tube meets the stomach.

1 Dec 2022

Rogue killer T cells associated with leukemia drive autoimmune diseases

Gene variants associated with leukemia can produce 'rogue' immune cells that drive autoimmune diseases, according to a new study from the Garvan Institute of Medical Research.

28 Nov 2022

Researchers discover a new function of CRISPR/Cas9 gene scissors

For several years now, the CRISPR/Cas9 gene scissors have been causing a sensation in science and medicine.

25 Nov 2022

New CRISPR-based tool can snip out faulty genes and replace them with new ones

Building on the CRISPR gene-editing system, MIT researchers have designed a new tool that can snip out faulty genes and replace them with new ones, in a safer and more efficient way.

24 Nov 2022

Discovery could lead to new mantle cell lymphoma drugs

A form of blood cancer known as mantle cell lymphoma is critically dependent on a protein that coordinates gene expression, such that blocking its activity with an experimental drug dramatically slows the growth of this lymphoma in preclinical tests, according to a study from Weill Cornell Medicine researchers.

22 Nov 2022

MIT researchers develop new technique for genome-wide studies of the liver

The liver’s ability to regenerate itself is legendary. Even if more than 70 percent of the organ is removed, the remaining tissue can regrow an entire new liver.

21 Nov 2022

Study reveals mechanism by which malignant cells shut down anti-cancer immune responses

A Ludwig Cancer Research study has revealed a single protein expressed at high levels by cancer cells across a broad range of malignancies that erects a multifaceted barrier to anti-cancer immune responses in mouse models of cancer and so shields tumors from immune detection and destruction.

20 Nov 2022

Treatment using CRISPR genome editing alleviates swelling attacks in hereditary angioedema patients

Hereditary angioedema (HAE) is a rare, genetic disorder characterized by severe, recurring, and unpredictable swelling attacks in various organs and tissues of the body, which can be painful, debilitating, and life-threatening.

10 Nov 2022

Scientists determine 5,072 essential human genes

In a recent study published in the journal Cell, researchers examined the phenotypic landscape of multiple elusive essential human genes to create a detailed genotype-phenotype resource outlining the phenotypic consequences of disrupting fundamental cellular processes.

8 Nov 2022

Novel gene editing therapy reduces circulating TTR protein levels in patients with ATTR amyloid cardiomyopathy

A single IV infusion of NTLA-2001, a novel CRISPR/Cas9-based gene editing therapy, significantly reduced circulating transthyretin (TTR) protein levels in patients with ATTR amyloid cardiomyopathy, a progressive and fatal cause of heart failure, according to late-breaking research presented today at the American Heart Association’s Scientific Sessions 2022.

7 Nov 2022

Study confirms the implication of Mib1 mutations in non-compaction cardiomyopathy

Non-compaction cardiomyopathy is a heart condition caused by defects that arise during fetal development and can have diverse health impacts in affected individuals, including sudden cardiac death.

7 Nov 2022

New control system could be used to finely tune the production of useful proteins

Using an approach based on CRISPR proteins, MIT researchers have developed a new way to precisely control the amount of a particular protein that is produced in mammalian cells.

1 Nov 2022

Specific stem cell protein found to be involved in the recurrence of medulloblastoma

The malignant brain tumor type medulloblastoma can become resistant to therapy which can cause relapse. Researchers at Uppsala University have discovered a certain protein that makes tumor cells resting and insensitive to radiation treatment.

28 Oct 2022

Researchers use CRISPR-edited T cells to treat seriously ill children with resistant leukaemia

Researchers at Great Ormond Street Hospital for Children (GOSH) and UCL Great Ormond Street Institute of Child Health (UCL GOS ICH) have used CRISPR/Cas9 technology to engineer donor T cells to try to treat seriously ill children with resistant leukaemia, who had otherwise exhausted all available therapies.

27 Oct 2022

Discovery points to a new understanding of Stargardt disease progression

Using a new stem-cell-based model made from skin cells, scientists found the first direct evidence that Stargardt-related ABCA4 gene mutations affect a layer of cells in the eye called the retinal pigment epithelium (RPE).