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.

Beneficial effects of gene therapy can be seen in SCID-X1 patients decades after treatment

An international collaboration between Great Ormond Street Hospital, the UCL GOS Institute for Child Health and Harvard Medical School has shown that the beneficial effects of gene therapy can be seen decades after the transplanted blood stem cells has been cleared by the body.

17 Mar 2021

UVA researcher developing innovative gene therapy to help children with Rett syndrome

A University of Virginia School of Medicine scientist is developing an innovative gene therapy she hopes will slow disease progression and improve movement, coordination and communication in children with Rett syndrome.

17 Mar 2021

New approach could treat infertility caused by abnormal GnRH secretion

Reports suggest that at least 25% of ovarian disorders are due to dysfunction of the brain mechanism in the hypothalamus that controls the gonadotropin-releasing hormone (GnRH), which is a molecule that governs the reproductive function in men and women by regulating the release of a range of reproductive hormones into the body, particularly gonadotropins, from the pituitary gland, which is located underneath the brain.

12 Mar 2021

Phase 2 clinical trial is now open at Columbia to test gene therapy for dry macular degeneration

A phase 2 clinical trial is underway at Columbia University and NewYork-Presbyterian to test an investigational gene therapy for dry age-related macular degeneration, a progressive, chronic eye disease with no cure and a leading cause of blindness in the United States.

9 Mar 2021

New academic spin-out based in Finland introduces a nasal spray vaccine against COVID

Rokote Laboratories Finland Ltd., a newly-founded academic spin-out based in Finland is working to develop and introduce to the markets a nasal spray vaccine against COVID.

8 Mar 2021

Discovery offers a precise route to treat inflammation associated with neuropathic pain

One of the hallmarks of chronic pain is inflammation, and scientists at the UNC School of Medicine have discovered that anti-inflammatory cells called MRC1+ macrophages are dysfunctional in an animal model of neuropathic pain.

5 Mar 2021

PICI awards $3 million to support early career cancer researchers

From improving immunotherapy response in childhood cancer patients, to better understanding how cancer's spread to the liver affects the body's immune system, to helping T cells stay energized to fight cancer, The Parker Institute for Cancer Immunotherapy (PICI)'s 2021 class of early career researchers award recipients are pursuing ideas that have the potential to change the face of oncology.

3 Mar 2021

ACGT awards $500,000 to advance immunotherapy approach for treating solid tumors

A scientific team led by Brian Brown, PhD, Professor of Genetics and Genomic Sciences and Associate Director of the Precision Immunology Institute at the Icahn School of Medicine at Mount Sinai (New York, New York), has been awarded $500,000 from Alliance for Cancer Gene Therapy to advance a promising immunotherapy approach to fight lung cancer and other solid tumor cancers.

1 Mar 2021

Catalent to Acquire Delphi Genetics and Launch US Plasmid Manufacturing Site to Establish Global pDNA Development and Manufacturing Capabilities

Catalent and Delphi Genetics today announced that they have entered into a final and definitive agreement whereby Catalent will acquire 100% of the shares of Delphi Genetics.

26 Feb 2021

Catalent to Acquire Delphi Genetics and Launch US Plasmid Manufacturing Site to Establish Global pDNA Development and Manufacturing Capabilities

Catalent and Delphi Genetics today announced that they have entered into a final and definitive agreement whereby Catalent will acquire 100% of the shares of Delphi Genetics.

26 Feb 2021

Canadian pilot study uses gene therapy to successfully treat patients with Fabry disease

Results of a world-first Canadian pilot study on patients treated with gene therapy for Fabry disease show that the treatment is working and safe.

25 Feb 2021

A new combination therapy for treating rare, fatal Sanfilippo syndrome in children

A study aiming to develop a new therapeutic technique could bring a revolution in our approach to treating rare, fatal Sanfilippo syndrome, a disorder that affects children as young as 2 years old and leads to childhood dementia and premature death.

24 Feb 2021

Novel vectors reduce the risks associated with ocular gene therapies

Strategies based on the use of gene therapy to mitigate the effects of mutations that cause blindness are undergoing rapid development.

22 Feb 2021

UC San Diego researchers launch clinical trial to test gene therapy for Alzheimer’s disease

Researchers at University of California San Diego School of Medicine have launched a first-in-human Phase I clinical trial to assess the safety and efficacy of a gene therapy to deliver a key protein into the brains of persons with Alzheimer's disease (AD) or Mild Cognitive Impairment (MCI), a condition that often precedes full-blown dementia.

18 Feb 2021

Researchers find a way to improve deletion efficiency of new genome-editing tool

CRISPR deletion (CRISPR-del) is a new genome editing tool that can delete or cut out certain pieces of DNA in living cells with surgical precision.

17 Feb 2021

Biodegradable microcapsules loaded with nerve growth factor can guide neuronal development

Researchers from Skoltech and their colleagues have demonstrated that nanoengineered biodegradable microcapsules can guide the development of hippocampal neurons in an in vitro experiment.

16 Feb 2021

Research shows how chemicals in the tumor microenvironment enable cancer to evade attack

A paper published today in Nature shows how chemicals in the areas surrounding tumors--known as the tumor microenvironment--subvert the immune system and enable cancer to evade attack. These findings suggest that an existing drug could boost cancer immunotherapy.

15 Feb 2021

$FDA approves cell-based gene therapy to treat adults with relapsed or refractory large-B-cell lymphoma$

FDA approves cell-based gene therapy to treat adults with relapsed or refractory large-B-cell lymphoma

Today, the U.S. Food and Drug Administration approved Breyanzi (lisocabtagene maraleucel), a cell-based gene therapy to treat adult patients with certain types of large B-cell lymphoma who have not responded to, or who have relapsed after, at least two other types of systemic treatment.

5 Feb 2021

Targeted pulses of ultrasound can help treat a range of brain diseases

Ultrasound is not only used as an imaging technique but targeted pulses of ultrasound can be used as a highly accurate treatment for a range of brain diseases, for which there were previously only limited treatment options.

5 Feb 2021

Spark Therapeutics launches gene therapy clinical trial for late-onset Pompe disease

Pompe disease is a hereditary genetic disorder caused by a deficiency of acid alpha-glucosidase leading to build-up of glycogen in the lyosomes, which then causes cell damage in various tissues, in particular the heart, the muscles, the liver and the nervous system.

3 Feb 2021

Gene Therapy News and Research

Further Reading

Beneficial effects of gene therapy can be seen in SCID-X1 patients decades after treatment

UVA researcher developing innovative gene therapy to help children with Rett syndrome

New approach could treat infertility caused by abnormal GnRH secretion

Phase 2 clinical trial is now open at Columbia to test gene therapy for dry macular degeneration

New academic spin-out based in Finland introduces a nasal spray vaccine against COVID

Discovery offers a precise route to treat inflammation associated with neuropathic pain

PICI awards $3 million to support early career cancer researchers

ACGT awards $500,000 to advance immunotherapy approach for treating solid tumors

Catalent to Acquire Delphi Genetics and Launch US Plasmid Manufacturing Site to Establish Global pDNA Development and Manufacturing Capabilities

Catalent to Acquire Delphi Genetics and Launch US Plasmid Manufacturing Site to Establish Global pDNA Development and Manufacturing Capabilities

Canadian pilot study uses gene therapy to successfully treat patients with Fabry disease

A new combination therapy for treating rare, fatal Sanfilippo syndrome in children

Novel vectors reduce the risks associated with ocular gene therapies

UC San Diego researchers launch clinical trial to test gene therapy for Alzheimer’s disease

Researchers find a way to improve deletion efficiency of new genome-editing tool

Biodegradable microcapsules loaded with nerve growth factor can guide neuronal development

Research shows how chemicals in the tumor microenvironment enable cancer to evade attack

FDA approves cell-based gene therapy to treat adults with relapsed or refractory large-B-cell lymphoma

Targeted pulses of ultrasound can help treat a range of brain diseases

Spark Therapeutics launches gene therapy clinical trial for late-onset Pompe disease

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