Gene Editing News and Research

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Adenine base editors show potential to correct X-CGD-causing mutations

A collaborative effort between investigators at the National Institutes of Health's National Institute of Allergy and Infectious Diseases (NIAID) and Massachusetts General Hospital (MGH), a founding member of the Mass General Brigham healthcare system, demonstrates the potential of precise genome editing technologies, called adenine base editors, to correct disease-causing mutations in stem cells from patients with X-linked chronic granulomatous disease (X-CGD), a rare genetic disorder characterized by high susceptibility to infections.

16 Oct 2024

Gene editing restores treatment sensitivity in melanoma

In a potential advance for melanoma patients, researchers at ChristianaCare’s Gene Editing Institute have used CRISPR gene editing tools to disable a gene mutation often seen in aggressive forms of this dangerous skin cancer that renders promising treatments ineffective.

16 Oct 2024

Gene editing enhances immunotherapy success by preventing T-cell exhaustion

Immunotherapy, using a patient's own immune system to treat disease, has shown promise in some patients with cancer but has not worked in most.

12 Oct 2024

Grant supports gene-editing research for rare metabolic diseases

A $14M grant will fund research on gene-editing therapies for rare metabolic diseases at the Perelman School of Medicine at the University of Pennsylvania (Penn) and Children's Hospital of Philadelphia (CHOP).

10 Oct 2024

Overcoming CRISPR's side effects for therapeutic applications

CRISPR is a revolutionary tool that allows scientists to precisely modify the genome and gene expression of cells in any organism. It's a reagent - a substance that facilitates a reaction - that combines an enzyme with a programmable RNA capable of locating specific genetic sequences. Once guided to the correct spot, the enzyme acts like a pair of scissors, cutting, replacing, or deleting sequences of DNA.

7 Oct 2024

Novel lipid nanoparticles enhance tissue-specific drug delivery

Penn Engineers have discovered a novel means of directing lipid nanoparticles (LNPs), the revolutionary molecules that delivered the COVID-19 vaccines, to target specific tissues, presaging a new era in personalized medicine and gene therapy.

1 Oct 2024

ERS Genomics and Université de Montréal sign CRISPR/Cas9 license agreement

Université de Montréal gains access to ERS Genomics' CRISPR/Cas9 patents, launching platforms for genome-wide screening and fostering research collaborations.

From ERS Genomics 1 Oct 2024

NIH grant supports research on cognitive decline in individuals with Lewy body diseases

Penn Medicine researchers will advance their understanding of the underlying causes of cognitive decline in individuals with Lewy body diseases, like Parkinson's, and identify biomarkers that predict which individuals are more likely to develop dementia with a $18 million grant from the National Institutes of Health/National Institute on Aging.

30 Sep 2024

Advanced brain mapping reveals neuronal subtypes and their connections

Scientists at the Salk Institute are unveiling a new brain-mapping neurotechnology called Single Transcriptome Assisted Rabies Tracing (START). The cutting-edge tool combines two advanced technologies - monosynaptic rabies virus tracing and single-cell transcriptomics - to map the brain's intricate neuronal connections with unparalleled precision.

30 Sep 2024

Patient-derived gut organoids reveal new insights into Crohn's disease subtypes

Crohn's disease - an autoimmune disorder - is characterized by chronic inflammation of the digestive tract, resulting in a slew of debilitating gastrointestinal symptoms that vary from patient to patient.

26 Sep 2024

CRISPR-Cas13 emerges as a game changer in RNA-targeted therapies

In recent years, the scientific community has made significant strides in the field of gene editing, particularly through the development of the CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) systems.

25 Sep 2024

The role of CRISPR-Cas9 in combating mosquito-borne diseases

Announcing a new article publication for Zoonoses journal. As global warming increases, mosquito activity areas are expanding, alongside changes in the natural environment and the misuse of insecticides, thus further increasing mosquito resistance and exposing the world to an elevated risk of mosquito-borne diseases.

23 Sep 2024

Enhanced TnpB protein offers new possibilities for gene editing

CRISPR-Cas is used broadly in research and medicine to edit, insert, delete or regulate genes in organisms. TnpB is an ancestor of this well-known "gene scissor" but is much smaller and thus easier to transport into cells.

23 Sep 2024

MaxCyte signs strategic platform license with Kamau Therapeutics to accelerate the development of cell therapies for genetic diseases

MaxCyte, Inc., a leading, cell-engineering focused company providing enabling platform technologies to advance the discovery, development and commercialization of next-generation cell-based therapeutics and innovative bioprocessing applications, and Kamau Therapeutics, a clinical-stage stem cell therapy gene correction company, today announced they are entering into a strategic platform license (SPL) agreement.

From MaxCyte, Inc. 23 Sep 2024

New discovery offers hope for overcoming CAR-T cell therapy resistance

Mayo Clinic researchers mined the molecular foundations of cancer and uncovered a new reason chimeric antigen receptor (CAR-T cell therapy) fails in some patients.

12 Sep 2024

VitelloTag approach offers new solution for gene editing challenges in egg cells

A new approach for delivering miniature research tools into the interior of egg cells and embryos has been developed at the Marine Biological Laboratory (MBL), resolving a major bottleneck to using the gene-editing tool CRISPR-Cas9 in many research organisms.

11 Sep 2024

A promising new therapeutic target for MASLD identified

Using a novel stem cell platform, a team of the Medical University of South Carolina researchers has identified a pathway that could be targeted by drugs to reduce fat accumulation in patients with a common form of fatty liver disease known as metabolic dysfunction-associated steatotic liver disease, or MASLD.

11 Sep 2024

Gene therapy shows dramatic vision improvement for rare inherited condition

The vision of people with a rare inherited condition that causes them to lose much of their sight early in childhood was 100 times better after they received gene therapy to address the genetic mutation causing it.

5 Sep 2024

ERS Genomics and medicines discovery catapult sign CRISPR/Cas9 license agreement

Dublin, Ireland, and Cheshire, UK, 04 September 2024: ERS Genomics Limited (‘ERS’), the CRISPR licensing Company, and Medicines Discovery Catapult (‘MDC’), an independent, not-for-profit drug discovery innovation center, have signed a non-exclusive commercial-use CRISPR license agreement.

From ERS Genomics 5 Sep 2024

Scientists advance type 1 diabetes treatment with cutting-edge stem cell and gene editing technologies

Researchers review significant advancements and ongoing challenges in cell replacement therapies for type 1 diabetes, highlighting innovative approaches in stem cell-derived islets and immune protection strategies.

5 Sep 2024