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.

Novel method to deliver drugs into inner ear restores hearing in deaf mice

An international team of researchers has developed a new method to deliver drugs into the inner ear. The discovery was possible by harnessing the natural flow of fluids in the brain and employing a little understood backdoor into the cochlea.

28 Jun 2023

Cell and gene therapy CDMO Andelyn Biosciences celebrates over 20 years of leadership and innovation with opening of new 180,000 square foot manufacturing headquarters

Andelyn Biosciences, a pioneer in the development and manufacturing of cell and gene therapies, today formally inaugurates its new manufacturing headquarters, the Andelyn Corporate Center (ACC), located in the Innovation District in Columbus, Ohio.

From Andelyn Biosciences 23 Jun 2023

World's largest gathering of NF community convenes in Arizona to advance research and care

From June 21 through June 27, the largest gathering of NF researchers, clinicians, and patients in the world will take place at the Fairmont Scottsdale Princess in Scottsdale, Arizona.

22 Jun 2023

Newborn screening for "bubble-baby" disease boosts survival of children

Screening newborns for severe combined immunodeficiency disease (SCID) significantly increases the survival of children after bone marrow transplantation, a new North American study finds.

21 Jun 2023

Newborn screening facilitates early identification of infants with severe combined immunodeficiency

Introducing widespread screening of newborns for a deadly disease called severe combined immunodeficiency, or SCID, followed by early treatment boosted the five-year survival rate of children with the disorder from 73% before the advent of screening to 87% since, researchers report.

20 Jun 2023

Phase I clinical study of TCR NK cell therapy for sarcoma expected to commence later this year

The University of Texas MD Anderson Cancer Center and Replay today announced that the Food & Drug Administration (FDA) has issued a 'safe to proceed' for the Investigational New Drug (IND) application for NY-ESO-1 TCR/IL-15 NK, an engineered T cell receptor natural killer (TCR NK) cell therapy for advanced synovial sarcoma and myxoid/round cell liposarcoma.

20 Jun 2023

Catalent adds new cryogenic capabilities at Shiga, Japan, facility to support clinical supply demand for cell and gene therapy development

Catalent today announced that it has expanded the services and capabilities at its facility in Shiga, Japan, to include the storage, kitting, and distribution of advanced therapies at ultra-low temperatures for clinical trials.

From Catalent 16 Jun 2023

New base-edited T-cells proven effective to treat lymphoblastic leukemia

Three young patients with relapsed T-cell leukemia have now been treated with base-edited T-cells, as part of a 'bench-to-bedside' collaboration between UCL and Great Ormond Street Hospital for Children (GOSH).

15 Jun 2023

Charles River Laboratories Launches Off-the-Shelf Lentiviral Vector Packaging Plasmids

Charles River Laboratories International, Inc. (NYSE: CRL) today announced the launch of its off-the-shelf lentiviral vector (LVV) packaging plasmids, providing a streamlined solution to the challenges associated with plasmid sourcing in cell and gene therapies.

From Charles River Laboratories, Inc. 13 Jun 2023

Study may help advance data on oncolytic adenovirus mediated double suicide-gene therapy for high-grade brain tumors

Researchers in the Hermelin Brain Tumor Center at Henry Ford Health are leading a Phase I clinical trial studying the maximum tolerated dose of an oncolytic adenovirus, a mutated virus engineered to selectively replicate in and destroy cancer tissue, in combination with fractionated stereotactic radiosurgery among patients who are undergoing resection of a recurrent high-grade astrocytoma brain tumor.

8 Jun 2023

Single dose of anti-Müllerian hormone gene therapy can induce long-term contraception in domestic cats

Currently there are no contraceptives capable of producing permanent sterilization in companion animals. Spaying, the surgical removal of the ovaries and uterus, is the most widely used strategy to control unwanted reproduction in female cats.

6 Jun 2023

Therapeutic nanocarriers engineered from adult skin cells show promise for treating lung injury

Therapeutic nanocarriers engineered from adult skin cells can curb inflammation and tissue injury in damaged mouse lungs, new research shows, hinting at the promise of a treatment for lungs severely injured by infection or trauma.

6 Jun 2023

Cure Mito Foundation releases the first-of-its-kind online resource about Leigh syndrome

The Cure Mito Foundation, a parent-led organization dedicated to advancing research and treatments for Leigh syndrome, has launched the first-of-its-kind online resource about Leigh syndrome, the most common type of pediatric mitochondrial disease.

5 Jun 2023

New insights into how axons grow could lead to effective therapies for blindness, paralysis

Injuries to the nerves can blind or paralyze because adult nerve cells don't regenerate their connections. Now, a team of UConn School of Medicine researchers report in Development that at least a small population of nerve cells exist in everyone that could be coaxed to regrow, potentially restoring sight and movement.

31 May 2023

Experimental decoy provides long-term protection from SARS-Cov-2 infection

An experimental "decoy" provided long-term protection from infection by the pandemic virus in mice, a new study finds.

30 May 2023

Using ex vivo gene therapy to fight obesity and diabetes in mice

A scientific team from the University of Barcelona and the CIBERobn has designed a strategy to fight obesity and diabetes in mice through ex vivo gene therapy which consists of implanting cells that have been manipulated and transformed in order to treat a disease.

30 May 2023

Gene Therapy News and Research

Further Reading

Novel method to deliver drugs into inner ear restores hearing in deaf mice

Cell and gene therapy CDMO Andelyn Biosciences celebrates over 20 years of leadership and innovation with opening of new 180,000 square foot manufacturing headquarters

World's largest gathering of NF community convenes in Arizona to advance research and care

Newborn screening for "bubble-baby" disease boosts survival of children

Newborn screening facilitates early identification of infants with severe combined immunodeficiency

Phase I clinical study of TCR NK cell therapy for sarcoma expected to commence later this year

Catalent adds new cryogenic capabilities at Shiga, Japan, facility to support clinical supply demand for cell and gene therapy development

New base-edited T-cells proven effective to treat lymphoblastic leukemia

Charles River Laboratories Launches Off-the-Shelf Lentiviral Vector Packaging Plasmids

Study may help advance data on oncolytic adenovirus mediated double suicide-gene therapy for high-grade brain tumors

Single dose of anti-Müllerian hormone gene therapy can induce long-term contraception in domestic cats

Therapeutic nanocarriers engineered from adult skin cells show promise for treating lung injury

Cure Mito Foundation releases the first-of-its-kind online resource about Leigh syndrome

New insights into how axons grow could lead to effective therapies for blindness, paralysis

Experimental decoy provides long-term protection from SARS-Cov-2 infection

Using ex vivo gene therapy to fight obesity and diabetes in mice

Virally mediated gene therapy could potentially treat genetic hearing loss

Nonhuman primate study helps explain how stem cell transplantation can kill HIV virus

Bridging the Gap between Academia and Industry: The Power of Collaboration in Life Sciences

Study provides data to support gene supplementation as a treatment for autosomal dominant retinitis pigmentosa

Discover how SciY empowers scientists

Why use light scattering to analyze proteins and viral vectors?

Changing the landscape of R&D to build clinical success from the ground up

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