Gene Therapy News and Research

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Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery.

Top pharmaceutical company evaluates Optimer for precision liver medicine

Optimer delivery vehicle has been shipped to a pharma partner for external evaluation.

From Aptamer Group 22 Apr 2024

Researchers receive NIH grant to help develop gene therapy for HIV

Researchers at Washington University School of Medicine in St. Louis have received a $6.2 million grant from the National Institutes of Health (NIH) to develop a gene therapy that would modify the immune system's B cells to spur them to produce broadly neutralizing antibodies against HIV.

18 Apr 2024

New platform can deliver gene therapy precisely to specific areas of the brain

A novel approach to gene therapy is improving lives in ways once thought impossible. Researchers at The Ohio State University Wexner Medical Center and College of Medicine have developed a new platform to deliver the gene therapy precisely to specific areas of the brain.

18 Apr 2024

Targeted nanoparticle delivery of FZD4 decreases lung tumor growth and metastasis

A recent breakthrough study from the lab of Tanya Kalin, MD, PhD, professor of Child Health and Internal Medicine at the University of Arizona College of Medicine – Phoenix, has shown potential to improve therapeutic outcomes for patients suffering from lung cancers.

16 Apr 2024

Real-world data validates gene therapy for spinal muscular atrophy

Findings from a new study in the Journal of Neuromuscular Diseases, published by IOS Press, demonstrate the effectiveness of disease-modifying treatments (DMTs) in infants with spinal muscular atrophy (SMA).

15 Apr 2024

Bio-Techne surpasses 10,000 peer-reviewed publications citing RNAscope technology

Bio-Techne Corporation today announced that Advanced Cell Diagnostics (ACD), part of Bio-Techne's Spatial Biology Division, has surpassed 10,000 peer-reviewed publications referencing the use and application of RNAscope™ ISH technology.

From Bio-Techne 9 Apr 2024

Adeno-associated virus: The gene therapy revolution faces manufacturing and safety hurdles

Recombinant adeno-associated virus (rAAV)-based genetic applications to treat human diseases.

5 Apr 2024

Gene therapy and glycoside drugs offer new hope for polycystic kidney disease treatment

Researchers have shown that dangerous cysts, which form over time in polycystic kidney disease (PKD), can be prevented by a single normal copy of a defective gene.

4 Apr 2024

Lurie Children's Hospital administers first gene therapy for Duchenne muscular dystrophy in Illinois

On March 27, 2024, Ann & Robert H. Lurie Children's Hospital of Chicago treated its first patient with ELEVIDYS (delandistrogene moxeparvovec-rokl), the first gene therapy for Duchenne muscular dystrophy – a rare, genetic disease characterized by progressive muscle damage and weakness.

3 Apr 2024

Bio-Rad launches first ultrasensitive multiplexed digital PCR assay for breast cancer mutation detection in clinical research

Bio-Rad Laboratories, Inc., a global leader in life science research and clinical diagnostic products, today announced the launch of its first ultrasensitive multiplexed digital PCR assay, the ddPLEX ESR1 Mutation Detection Kit.

From Bio-Rad Laboratories 3 Apr 2024

New genetic system analyzes the underlying mechanisms of CRISPR-based DNA repair outcomes

Since its breakthrough development more than a decade ago, CRISPR has revolutionized DNA editing across a broad range of fields.

26 Mar 2024

Viroclinics-DDL changes name to Cerba Research

Viroclinics-DDL a world-leading vaccine and antiviral specialty laboratory providing research and clinical trial solutions rooted in virology, proudly announces changing its name to Cerba Research as of the 1st of April 2024.

From Cerba Research 26 Mar 2024

Researchers identify mechanism behind COVID-19 related brain fog

Among the many confounding symptoms in patients recovering from a COVID-19 infection are memory loss and difficulty learning.

21 Mar 2024

Investigational gene therapy shows promise for rare childhood neurodegenerative disease

An investigational gene therapy for a rare neurodegenerative disease that begins in early childhood, known as giant axonal neuropathy (GAN), was well tolerated and showed signs of therapeutic benefit in a clinical trial led by the National Institutes of Health.

20 Mar 2024

Hearing loss linked to accelerated Alzheimer's progression via GDF1 pathway

Study unveils the GDF1 signaling pathway's role in mitigating the adverse effects of hearing loss on Alzheimer's disease, spotlighting potential avenues for therapeutic development.

20 Mar 2024

Viroclinics-DDL rebrands to Cerba Research

Viroclinics-DDL (referred to herein as Cerba Research, the Netherlands) a world leading vaccine and antiviral specialty laboratory providing research and clinical trial solutions rooted in virology, proudly announces changing its name to Cerba Research as of the 1st of April 2024.

18 Mar 2024

CHOP study uncovers metabolic key to faster immune response

Researchers from Children's Hospital of Philadelphia identified a key metabolite in cells that helps direct immune responses and explains at a single cell level why immune cells that most efficiently recognize pathogens, vaccines, or diseased cells grow and divide faster than other cells.

15 Mar 2024

Scientists transform skin cells into functional eggs in mice

New research from Oregon Health & Science University describes the science behind a promising technique to treat infertility by turning a skin cell into an egg that is capable of producing viable embryos.

9 Mar 2024

Breakthrough in CRISPR delivery promises safer gene editing

Recent research in PNAS highlights advancements in non-viral, cell-specific delivery methods for CRISPR-Cas systems, emphasizing their potential to enhance research and gene therapy applications by minimizing risks associated with off-target effects and improving targeted delivery.

7 Mar 2024

Higher COVID-19 death risk found in sickle cell disease and trait patients

UdeM's Stéphanie Forté and co-researchers in Canada and Brazil have shown how people with sickle cell disease or sickle cell trait have a higher chance of dying from COVID-19 than the general population.

29 Feb 2024