Gene Editing News and Research

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Gene editing with CRISPR may introduce new defects

The CRISPR tool is capable of repairing the genetic defect responsible for the immune disease chronic granulomatous disease.

6 Nov 2024

New therapeutic target overcomes ovarian cancer immunotherapy resistance

Researchers at the Icahn School of Medicine at Mount Sinai have discovered a way that ovarian cancer tumors manipulate their environment to resist immunotherapy and identified a drug target that could overcome that resistance.

1 Nov 2024

Researchers uncover key mechanisms of CRISPR gene editing

CRISPR/Cas9 is a gene editing tool that has revolutionized biomedical research and led to the first FDA-approved CRISPR-based gene therapy.

28 Oct 2024

New research unveils mechanism behind deadly neuroendocrine prostate cancer

A leading scientist whose discoveries about prostate cancer led to lifesaving treatments is now shedding light on an emerging and deadlier form of the disease: neuroendocrine prostate cancer (NEPC).

28 Oct 2024

Global leaders gather at the NFCR Summit to drive breakthroughs in cancer care

The 2024 National Foundation for Cancer Research (NFCR) Global Summit and Award Ceremonies for Cancer Research & Entrepreneurship, co-hosted with the AIM-HI Accelerator Fund, convened the world's top experts across cancer research, biotech entrepreneurship, pharmaceuticals, investment, and patient advocacy.

28 Oct 2024

Unlocking the secrets of Usher syndrome

The mistakes are small but carry severe consequences: In Usher syndrome, genetic mutations cause both hearing and vision loss. Cell biologist Professor Uwe Wolfrum from Mainz University is researching the molecular background of this rare genetic disorder - supported by foundations and, not least, by affected individuals themselves

28 Oct 2024

Single treatment with CRISPR gene editing therapy offers hope for hereditary angioedema patients

A single treatment with, a CRISPR-Cas9 based gene editing therapy, is enough to replace the daily medication of patients with hereditary angioedema (HAE), a condition characterized by severe, painful and sudden onset of swelling, sometimes resulting in death.

25 Oct 2024

Human midbrain organoids: A promising tool in the fight against Parkinson's disease

Advancements in organoid technology are crucial for Parkinson's disease research, providing human-relevant models for studying disease and testing therapies.

23 Oct 2024

Scientists reveal how miRNAs shape cancer and offer new paths for treatment

Review explores how microRNAs (miRNAs) interact in cancer development, their potential in diagnosis, and innovative therapeutic strategies, including overcoming chemotherapy resistance.

20 Oct 2024

Researchers develop strategies to identify regulators of intestinal hormone secretion

A multi-institutional group of researchers led by the Hubrecht Institute and Roche's Institute of Human Biology has developed strategies to identify regulators of intestinal hormone secretion.

18 Oct 2024

Scientists map the genetic landscape of drug resistance in cancers

All cancer mutations that cause drug resistance fall into one of four categories. New research has detailed each type, helping to uncover targets for drug development and identify potential effective second-line therapies.

18 Oct 2024

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