Gene Editing News and Research

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Customized CRISPR gene editing therapy successfully treats infant with rare disease

In a historic medical breakthrough, a child diagnosed with a rare genetic disorder has been successfully treated with a customized CRISPR gene editing therapy by a team at Children's Hospital of Philadelphia (CHOP) and Penn Medicine.

15 May 2025

Mitochondrial DNA editing offers new hope for neurodegenerative disease treatment

Mitochondrial DNA (mtDNA) editing has emerged as a revolutionary approach in the fight against neurodegenerative diseases (NDDs).

12 May 2025

Researchers uncover how Entamoeba histolytica evades the immune system

The single-celled parasite Entamoeba histolytica infects 50 million people each year, killing nearly 70,000. Usually, this wily, shape-shifting amoeba causes nothing worse than diarrhea.

12 May 2025

Gene-edited immune cells show promise against advanced GI cancers

Researchers at the University of Minnesota have completed a first-in-human clinical trial testing a CRISPR/Cas9 gene-editing technique to help the immune system fight advanced gastrointestinal (GI) cancers.

3 May 2025

New gene editing tool enables precise control of mitochondrial mutation load

Mitochondrial diseases affect approximately 1 in 5,000 people worldwide, causing debilitating symptoms ranging from muscle weakness to stroke-like episodes.

3 May 2025

New era of medicine emerges through structural control of nanotherapeutics

Historically, the vast majority of pharmaceutical drugs have been meticulously designed down to the atomic level.

25 Apr 2025

Machine learning unlocks millions of safer CRISPR enzymes for gene editing

Genome editing has advanced at a rapid pace with promising results for treating genetic conditions-but there is always room for improvement.

22 Apr 2025

New study sheds light on 3D gene hubs driving brain cancer

The way DNA folds inside the nucleus of brain cells may hold the key to understanding a devastating form of brain cancer called glioblastoma, suggests a new preclinical study from Weill Cornell Medicine researchers.

22 Apr 2025

Boost your transfection success with these proven expert tips

Gain expert insights in this interview on achieving transfection success. Explore proven strategies to optimize your approach and enhance results.

From Sartorius 22 Apr 2025

iPS cell transplants show early promise for treating Parkinson's disease safely

In a first-of-its-kind human trial, researchers transplanted iPS-cell-derived dopaminergic progenitors into patients with Parkinson’s disease and observed promising motor improvements with no serious safety concerns over 24 months. The therapy showed increased dopamine synthesis without tumor formation or graft-induced dyskinesia.

21 Apr 2025

Engineered brain cells offer new hope for treating Alzheimer's

A new way to deliver disease-fighting proteins throughout the brain may improve the treatment of Alzheimer's disease and other neurological disorders, according to University of California, Irvine scientists.

21 Apr 2025

New therapeutic frontiers in the management of achondroplasia

A recent publication in Genes & Diseases has delivered a compelling synthesis of the latest insights into the cellular mechanisms and therapeutic interventions for achondroplasia, the most common form of genetic dwarfism.

20 Apr 2025

New enzyme family enables targeted cuts in single-stranded DNA

An INRS team discovers a new family of enzymes capable of inducing targeted cuts in single-stranded DNA .

16 Apr 2025

New STITCHR tool offers a promising step forward for gene therapy

Investigators from Mass General Brigham and Beth Israel Deaconess Medical Center have developed STITCHR, a new gene editing tool that can insert therapeutic genes into specific locations without causing unwanted mutations.

9 Apr 2025

Transforming genetic deafness treatment with base editing

Congenital hearing loss refers to impaired auditory function that occurs due to genetic causes. GJB2 is the gene responsible for approximately half of all cases of hereditary hearing loss.

27 Mar 2025

Researchers develop a reverse genetics system for African swine fever virus

Researchers from the J. Craig Venter Institute (JCVI), the Friedrich-Loeffler-Institut (FLI), and the International Livestock Research Institute (ILRI) have developed a reverse genetics system for African swine fever virus (ASFV).

26 Mar 2025

Groundbreaking study unveils key mechanism for neuronal identity regulation

A team of researchers from the Institute for Neurosciences, a joint center of the Spanish National Research Council (CSIC) and the Miguel Hernández University (UMH) of Elche, in collaboration with researchers from Columbia University (New York, USA), has identified a mechanism that regulates the production of two different proteins from the same gene.

22 Mar 2025