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.

ERS Genomics and Cellular Engineering Technologies Enter CRISPR/Cas9 License Agreement

ERS Genomics Limited and Cellular Engineering Technologies today announced a non-exclusive license agreement.

17 Aug 2021

Study identifies mechanism of resistance and presents a much-needed target for advanced leukemias

Too many "exhausted" T cells left in the wake of aggressive chemotherapy regimens for patients with advanced chronic lymphocytic leukemia (CLL) make it more challenging for chimeric antigen receptor (CAR) T cell therapy to do its job.

16 Aug 2021

Newborn screening and early treatment for spinal muscular atrophy can save both lives and money

Gene therapy for spinal muscular atrophy might have a high up-front price tag. But by screening and treating infants early, the therapy can save both lives and money in the long term.

4 Aug 2021

Biolabs LA at The Lundquist Institute reaches highly significant capacity milestone

BioLabs LA at The Lundquist Institute, a co-working and incubator facility for high-potential bioscience start-ups, has reached 100% capacity ahead of schedule.

3 Aug 2021

Start codon disruption with CRISPR-Cas9 gene editing prevents Fuchs’ corneal dystrophy in mice

In a new study, researchers at the University of Oregon's Phil and Penny Knight Campus for Accelerating Scientific Impact used start codon disruption with CRISPR-Cas9 gene editing to prevent Fuchs' corneal dystrophy in mice.

3 Aug 2021

Gene therapy protects optic nerve cells and preserves vision in glaucoma mouse models

A form of gene therapy protects optic nerve cells and preserves vision in mouse models of glaucoma, according to research supported by NIH's National Eye Institute.

23 Jul 2021

Investigational Oncomagnetic Device shrinks end-stage recurrent glioblastoma

Houston Methodist Neurological Institute researchers from the department of neurosurgery shrunk a deadly glioblastoma tumor by more than a third using a helmet generating a noninvasive oscillating magnetic field that the patient wore on his head while administering the therapy in his own home.

22 Jul 2021

Chemical modification in RNA molecules may offer protection against liver disease

A chemical modification that occurs in some RNA molecules as they carry genetic instructions from DNA to cells' protein-making machinery may offer protection against non-alcoholic fatty liver, a condition that results from a build-up of fat in the liver and can lead to advanced liver disease, according to a new study by UCLA researchers.

19 Jul 2021

Gene therapy delivered into the brain may be safe, effective in treating rare neurological disorder

A new study published in Nature Communications suggests that gene therapy delivered into the brain may be safe and effective in treating aromatic L-amino acid decarboxylase (AADC) deficiency.

16 Jul 2021

Researchers study the role of subnuclear NSrp70 in T cell-mediated adaptive immunity

T cells are components of the body’s immune system. The ability of T cells to recognize diverse pathogens and mount a defense against them stems from the way proteins are spliced during their development and maturation.

15 Jul 2021

Yale School of Public Health and Tevogen Bio partner to expand school's regulatory sciences program

The Yale School of Public Health is partnering with Tevogen Bio, a clinical stage biotechnology company, on a major expansion of the school's regulatory sciences program.

14 Jul 2021

Curia to Acquire US-based LakePharma, Expanding End-to-End R&D Capabilities and Scale for Biologics

Curia today announced it has entered into a definitive agreement to acquire LakePharma Inc.

From Curia 14 Jul 2021

Gene therapy shows promise in children with devastating genetic disorder

Children with a devastating genetic disorder characterized by severe motor disability and developmental delay have experienced sometimes dramatic improvements in a gene therapy trial launched at UCSF Benioff Children's Hospitals.

12 Jul 2021

Novel method of gene therapy promises to transform the treatment of rare genetic disorder

A novel method of gene therapy is helping children born with a rare genetic disorder called AADC deficiency that causes severe physical and developmental disabilities.

12 Jul 2021

Study: Gene editing therapy for early-stage Huntington’s disease may have long-lasting benefits

In a new study on mice, Johns Hopkins Medicine researchers report that using MRI scans to measure blood volume in the brain can serve as a noninvasive way to potentially track the progress of gene editing therapies for early-stage Huntington's disease, a neurodegenerative disorder that attacks brain cells.

7 Jul 2021

Nanotechnology and the fight against COVID-19

In a review paper published in the open-access MDPI journal Viruses, a team of researchers explored the potential of nanotechnology to alleviate the current and future coronavirus burden. The team focused on its potential role in the detection, diagnosis, protection, and treatment of SARS-CoV-2 infection.

29 Jun 2021

Preliminary results of AAV-mediated gene therapy for Crigler Najjar syndrome

Preliminary results from the European gene therapy trial for Crigler-Najjar syndrome, conducted by Genethon in collaboration with European network CureCN, were presented at the EASL (European Association for the Study of the Liver) annual International Liver Congress on June 26.

28 Jun 2021

Gene Therapy News and Research

Further Reading

ERS Genomics and Cellular Engineering Technologies Enter CRISPR/Cas9 License Agreement

Study identifies mechanism of resistance and presents a much-needed target for advanced leukemias

Newborn screening and early treatment for spinal muscular atrophy can save both lives and money

Biolabs LA at The Lundquist Institute reaches highly significant capacity milestone

Start codon disruption with CRISPR-Cas9 gene editing prevents Fuchs’ corneal dystrophy in mice

Gene therapy protects optic nerve cells and preserves vision in glaucoma mouse models

Investigational Oncomagnetic Device shrinks end-stage recurrent glioblastoma

Chemical modification in RNA molecules may offer protection against liver disease

Gene therapy delivered into the brain may be safe, effective in treating rare neurological disorder

Researchers study the role of subnuclear NSrp70 in T cell-mediated adaptive immunity

Yale School of Public Health and Tevogen Bio partner to expand school's regulatory sciences program

Curia to Acquire US-based LakePharma, Expanding End-to-End R&D Capabilities and Scale for Biologics

Gene therapy shows promise in children with devastating genetic disorder

Novel method of gene therapy promises to transform the treatment of rare genetic disorder

Study: Gene editing therapy for early-stage Huntington’s disease may have long-lasting benefits

Nanotechnology and the fight against COVID-19

Preliminary results of AAV-mediated gene therapy for Crigler Najjar syndrome

RDM Positive Impact Foundation funds $1.25 million research project to study SYNGAP1

Researchers design a smart device that delivers medications to prevent HIV transmission

Repressing a protein that facilitates DNA repair to potentiate chemotherapy

Event guide: 9th Gene Therapy Development Summit 2026 eBook

The Critical Role of Sample Storage in Drug Discovery and Development

Advancing Neuroscience Research Through Collaboration and Patient-Centered Innovation

The impact of vibration on microscopy

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