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.

Revolutionary gene replacement surgery restores vision in patients with retinal degeneration

More than a year after becoming one of the first medical institutions nationally to complete a revolutionary gene replacement surgery to restore vision in patients with retinal degeneration, surgeons at the of The Vision Center at Children's Hospital Los Angeles have successfully completed the procedure on an additional 13 patients.

14 Jul 2019

Retina can restructure itself following gene therapy

Following gene therapy, the retina can restructure itself and regain normal light responses, according to research in mice published in JNeurosci.

From Society for Neuroscience 10 Jul 2019

ISTH introduces new global education initiative in gene therapy for hemophilia

The International Society on Thrombosis and Haemostasis is pleased to announce the official launch of Gene Therapy in Hemophilia: An ISTH Education Initiative.

8 Jul 2019

Schwann cells capable of generating protective myelin over nerves finds research

Researchers have found that Schwann cells, a special type of cells are capable of generating protective myelin sheath covers for the nerves at rates higher than earlier believed.

7 Jul 2019

Researchers completely eliminate all traces of HIV from infected mice

For the first time, researchers have successfully removed HIV-1 DNA from the genomes of living animals, a critical step towards a potential cure for the disease.

4 Jul 2019

Sartorius launches new ambr 250 modular bioreactor vessel for cell and gene therapy applications

Sartorius Stedim Biotech, a leading international partner of the biopharmaceutical industry, today launched a new vessel for its ambr 250 modular benchtop automated mini bioreactor system.

From Sartorius 28 Jun 2019

Researchers use genetically manipulated virus to combat prostate cancer

Researchers at the São Paulo State Cancer Institute in Brazil have succeeded in using a genetically manipulated virus to destroy tumor cells upon injection into mice with prostate cancer.

27 Jun 2019

Family Doctors play key role in imparting health education on advanced therapies

A recent study conducted jointly by the Tissue Engineering Research Group of the Department of Histology and the Family Medicine Unit of the University of Granada has highlighted the conceptual, attitudinal, and procedural profile of resident hospital doctors specializing in Family Medicine, in relation to the so-called advanced therapies.

25 Jun 2019

Study reveals genetic signature of failed immune response to cancer

Our immune system is programmed to destroy cancer cells. Sometimes it has trouble slowing disease progression because it doesn't act quickly or strongly enough.

21 Jun 2019

Scientists pinpoint cause of fatal respiratory disorder that turns babies' lips and skin blue

Scientists used a gene editing method called CRISPR/Cas9 to generate mice that faithfully mimic a fatal respiratory disorder in newborn infants that turns their lips and skin blue. The new laboratory model allowed researchers to pinpoint the ailment's cause and develop a potential and desperately needed nanoparticle-based treatment.

19 Jun 2019

Three signs of real progress against sickle cell disease

A decade ago, the United Nations declared June 19 to be World Sickle Cell Awareness Day- and for good reason.

19 Jun 2019

Henry Ford Cancer Institute treats its first patient with CAR T-cell therapy

Henry Ford Cancer Institute has treated its first patient with CAR T-cell therapy, an approach that uses engineered cells from a patient's immune system to destroy cancer.

18 Jun 2019

Complement system shown to remove dead cells in retinitis pigmentosa, contradicting previous research

Researchers have found that the complement system seems to protect against retinal degeneration in retinitis pigmentosa.

18 Jun 2019

A beginner’s guide to better understand curative and transformative therapies

Value in Health, the official journal of ISPOR, the professional society for health economics and outcomes research, announced today the publication of a series of articles offering important insight regarding the current state of curative and transformative therapies.

17 Jun 2019

New implant technology safely minimizes injections for people with macular degeneration

In a clinical trial of 220 people with “wet” age-related macular degeneration, Johns Hopkins Medicine researchers, collaborators from many sites across the country, and Genentech in South San Francisco have added to evidence that using a new implant technology that continuously delivers medication into the eyes is safe and effective in helping maintain vision and reduces the need for injections in the eyes.

From Johns Hopkins Medicine 14 Jun 2019

More screening and new treatments needed for sickle cell disease

The promise of new treatments for sickle cell disease, a group of inherited red blood cell disorders, has never been so great, but it will only be realized if there is constant investment in health policies such as programs for screening newborns, especially in sub-Saharan Africa, where most cases occur.

13 Jun 2019

New technology harnesses bacterial jumping genes for precise DNA integration

A new discovery by researchers at Columbia University Vagelos College of Physicians and Surgeons could fix one of the major shortcomings of current gene-editing tools, including CRISPR, and offer a powerful new approach for genetic engineering and gene therapy.

13 Jun 2019

URMC biotech start-up receives funding to develop cell-based therapies for neurological disorders

Oscine Therapeutics, a new biotechnology company based on discoveries made and developed at the University of Rochester Medical Center has received a significant multi-year investment to support both research and development of cell-based therapies for neurological disorders.

11 Jun 2019

Scientists successfully carry out gene editing in vivo

Scientists at Harvard University have successfully proven that stem cells can be edited inside the body, rather than in a petri dish.

5 Jun 2019

IDT releases new ultra-high performance CRISPR Cas12a enzyme

As an advocate for the needs of researchers working at the forefronts of genomic research, Integrated DNA Technologies releases its latest CRISPR enzyme Alt-R® Cas12a Ultra.

From Integrated DNA Technologies 31 May 2019