Gene Editing News and Research

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New map offers valuable insights into the underlying mechanisms of neurodevelopmental disorders and cancer

Scientists have created the first extensive map showing how all possible genetic changes can affect health in the gene DDX3X, leading to valuable insights into the underlying mechanisms of neurodevelopmental disorders and cancer.

11 Dec 2023

Many patients may not benefit from gene-editing treatments for sickle cell disease

Federal approval of a breakthrough gene-editing technology that treats the pain and debilitating effects of sickle cell disease is cause for celebration among a community with few options for relief.

8 Dec 2023

New study provides convincing evidence of the essential role of cylicins in sperm development and fertility

Researchers investigated the Role of Cylicins in Spermiogenesis and Male Fertility in Mice and Humans through Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/ CRISPR-associated protein 9 (Cas9) Gene Editing.

30 Nov 2023

Synthetic biologists develop versatile platform for targeted drug delivery

In recent years, cell and gene therapies have shown significant promise for treating cancer, cystic fibrosis, diabetes, heart disease, HIV/AIDS and other difficult-to-treat diseases.

27 Nov 2023

New stem cell-based treatment for Type 1 diabetes can reduce dependence on daily insulin injections

An innovative stem cell-based treatment for Type 1 diabetes can meaningfully regulate blood glucose levels and reduce dependence on daily insulin injections, according to new clinical trial results from the University of British Columbia (UBC) and Vancouver Coastal Health (VCH).

27 Nov 2023

New CRISPR approach shows promise for treating glioblastoma

The gene-editing technology CRISPR shows early promise as a therapeutic strategy for the aggressive and difficult-to-treat brain cancer known as primary glioblastoma, according to findings of a new study from Gladstone Institutes.

27 Nov 2023

Combined application of therapeutic viruses and nanomaterials to revolutionize cancer therapy

Using oncolytic viruses (OVs) in combination with nanomaterials as a novel approach to cancer treatment.

22 Nov 2023

Scientists uncover key to enhancing cancer-killing T cells

T cells are the key immune system component involved in killing cancer. Tumors produce signals that turn off these T cells, partially by forcing them to progressively differentiate (mature) into a hypofunctional state known as exhaustion.

21 Nov 2023

Rare APOE3-R136S mutation offers protection against Alzheimer's disease pathologies in new study

The R136S genetic mutation protected against apolipoprotein E4 (APOE4)-induced late-onset Alzheimer’s disease (LOAD).

16 Nov 2023

New approach to CAR T cell therapy may extend treatment effectiveness

CAR T cell therapy, a powerful type of immunotherapy, has begun to revolutionize cancer treatment. Pioneered at Memorial Sloan Kettering Cancer Center (MSK), the therapy involves engineering a patient's T cells so they recognize and attack cancer cells.

14 Nov 2023

"Christchurch mutation" can block detrimental effects of Alzheimer’s risk factor

Researchers at Gladstone Institutes have discovered that a rare genetic variant known as the "Christchurch mutation" can block detrimental effects of apolipoprotein E4, the best-established risk factor for the most common form of Alzheimer's disease.

13 Nov 2023

CRISPR-based gene-editing therapy could offer hope for people with inherited high LDL cholesterol

A single infusion of a CRISPR-based gene-editing therapy significantly reduced low-density lipoprotein cholesterol (LDL, the "bad cholesterol") in people who carry one gene for the inherited condition that results in very high LDL cholesterol levels and a high risk of heart attack at an early age, according to late-breaking science presented today at the American Heart Association's Scientific Sessions 2023.

13 Nov 2023

NanoSyrinx appoints Anthony Johnson as Non-Executive Director

Appointment of Big Pharma executive strengthens leadership team to drive ambitious growth strategy.

From NanoSyrinx 13 Nov 2023

UCI researchers discover gene critical for muscle repair

University of California, Irvine researchers have identified a gene expressed during regeneration that is critical for muscle repair.

12 Nov 2023

Gene therapy's double-edged sword: Breakthrough treatments face manufacturing and efficacy

Research explores the advancements, challenges, and economic implications of gene therapy for various diseases, including inherited blood disorders, malignancies treated with CAR-T cells, and diseases targeted by in vivo AAV vectors. The review highlights the transformative potential of gene therapies while acknowledging the complexities in manufacturing, safety, efficacy, and long-term durability.

12 Nov 2023

Time-lapsed images show how magnesium ions coordinate double-stranded breaks in CRISPR-Cas9

The gene-editing technology known as CRISPR has led to revolutionary changes in agriculture, health research and more.

2 Nov 2023

Gene editing breakthrough could offer new hope for people with phenylketonuria

Phenylketonuria (PKU) is a rare newborn genetic disease that impacts between 1 in 10,000 to 1 in 20,000 people, depending on the individuals' genetic ancestry. PKU causes an amino acid - called phenylalanine (Phe) - to build up in the bloodstream.

2 Nov 2023

Science, Innovation, and Xenotransplantation: A Discussion with Dr. Wenning Qin

In this interview, we delve deep into the pioneering world of xenotransplantation and the evolving landscape of gene editing.

2 Nov 2023

New gene editing method creates first mice with human-like COVID-19

Researchers have genetically engineered the first mice that get a human-like form of COVID-19, according to a study published online November 1 in Nature.

1 Nov 2023

How one gene shakes up our understanding of male infertility

The study investigates the role of the ACTL7B gene in sperm formation using genetically modified mice and finds that deficiencies in this gene lead to failed spermatogenesis and structural sperm abnormalities. Given the gene's similarity in humans, the findings suggest ACTL7B could serve as a potential biomarker for male infertility.

31 Oct 2023

Gene Editing News and Research

New map offers valuable insights into the underlying mechanisms of neurodevelopmental disorders and cancer

Many patients may not benefit from gene-editing treatments for sickle cell disease

New study provides convincing evidence of the essential role of cylicins in sperm development and fertility

Synthetic biologists develop versatile platform for targeted drug delivery

New stem cell-based treatment for Type 1 diabetes can reduce dependence on daily insulin injections

New CRISPR approach shows promise for treating glioblastoma

Combined application of therapeutic viruses and nanomaterials to revolutionize cancer therapy

Scientists uncover key to enhancing cancer-killing T cells

Rare APOE3-R136S mutation offers protection against Alzheimer's disease pathologies in new study

New approach to CAR T cell therapy may extend treatment effectiveness

"Christchurch mutation" can block detrimental effects of Alzheimer’s risk factor

CRISPR-based gene-editing therapy could offer hope for people with inherited high LDL cholesterol

NanoSyrinx appoints Anthony Johnson as Non-Executive Director

UCI researchers discover gene critical for muscle repair

Gene therapy's double-edged sword: Breakthrough treatments face manufacturing and efficacy

Time-lapsed images show how magnesium ions coordinate double-stranded breaks in CRISPR-Cas9

Gene editing breakthrough could offer new hope for people with phenylketonuria

Science, Innovation, and Xenotransplantation: A Discussion with Dr. Wenning Qin

New gene editing method creates first mice with human-like COVID-19

How one gene shakes up our understanding of male infertility

Industry focus eBook - Cell and gene therapy (2nd edition) eBook

Advancing Intracellular Delivery with LumiPore®

How ELRIG Is Helping Mid-Career Scientists Navigate Leadership, Career Change and Redundancy

The role of UV-Vis in biopharma QC

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