Gene Transfer News and Research

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Gene Transfer is the insertion of genetic material into a cell.

FDA advisory committee recommends Novartis' CAR-T therapy for young leukemia patients

Today the FDA's Oncologic Drugs Advisory Committee, an independent panel of experts, voted unanimously to recommend to the FDA approval of Novartis' experimental CAR-T therapy called Tisagenlecleucel, also known as CTL019.

13 Jul 2017

Viral vectors for gene transfer can travel to distant areas in the brain, study shows

Gene transfer is seen as a hopeful therapy for Alzheimer's and Parkinson's patients. The approach involves using harmless laboratory-produced viruses to introduce important genes into the brain cells.

16 Jun 2017

Researchers find long-lasting benefits of gene therapy in dog model of myotubular myopathy

Researchers who previously showed that a gene therapy treatment could save the lives of dogs with a deadly disease called myotubular myopathy—a type of muscular dystrophy that affects the skeletal muscles—have found that the therapy is long-lasting.

7 Jun 2017

Combining oral drug with personalized cell therapy can lead to complete remission in CLL patients

Combining the kinase inhibitor ibrutinib with an investigational personalized cellular therapy known as CTL119 can lead to complete remission in patients with high-risk chronic lymphocytic leukemia (CLL), according to new research from the Perelman School of Medicine at the University of Pennsylvania and Penn's Abramson Cancer Center

31 May 2017

Novel approach cures diabetes in mice for one year without side effects

A potential cure for Type 1 diabetes looms on the horizon in San Antonio, and the novel approach would also allow Type 2 diabetics to stop insulin shots.

5 May 2017

Meningitis bacterium appears to be adapting to urogenital environment, DNA analysis shows

Neisseria meningitidis, a bacterium usually associated with meningitis and sepsis, is the cause of a recent cluster of sexually transmitted infections in Columbus, Ohio and in other US cities. The bacterium appears to be adapting to a urogenital environment, an analysis of the organism's DNA shows.

4 Apr 2017

MSK researchers harness power of CRISPR/Cas9 to create more-potent CAR T cells

Researchers from Memorial Sloan Kettering Cancer Center have harnessed the power of CRISPR/Cas9 to create more-potent chimeric antigen receptor (CAR) T cells that enhance tumor rejection in mice.

23 Feb 2017

Vision scientists uncover reasons behind unusual perceptual properties of the eye's fovea

Vision scientists have uncovered some of the reasons behind the unusual perceptual properties of the eye's fovea. Only humans and other primates have this dimple-like structure in their retinas.

23 Feb 2017

Scientists restore partial hearing and balance in deaf mice using new gene therapy

Using a novel form of gene therapy, scientists from Harvard Medical School and the Massachusetts General Hospital have managed to restore partial hearing and balance in mice born with a genetic condition that affects both.

28 Jan 2017

New MIP system for therapeutic gene delivery can enhance transgene expression using AAV vector

Advanced engineering of a mini-intronic plasmid (MIP) system designed to carry a therapeutic gene can significantly enhance the expression of the transgene delivered using an adeno-associated viral (AAV) vector.

28 Jan 2017

Novel gene therapy restores partial hearing and balance in mice born with genetic deafness

27 Jan 2017

Study shows how phage-resistant cells become susceptible upon co-incubation with sensitive bacteria

Bacteriophages (phages) are probably the most abundant entities in nature, often exceeding bacterial densities by an order of magnitude.

11 Jan 2017

Orphan Disease Center establishes new initiative that focuses on ALS

The Orphan Disease Center in the Perelman School of Medicine at the University of Pennsylvania has established a new Program of Excellence for Motor Neuron Disease.

10 Jan 2017

AAV-based gene therapy to treat liver disorders advances into human testing

Liver-directed gene therapy delivered using adeno-associated viral (AAV) vectors to treat diseases such as hemophilia have advanced into human testing.

30 Dec 2016

Studies in non-human primates provide more predictive results in genetic causes of blindness

Many gene therapy-based approaches are in development to combat genetic and other causes of blindness and vision loss, and much can be learned about the safety and effectiveness of these promising new therapies by studying them first in non-human primates before initiating clinical trials, as shown by the results of a study published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers.

29 Dec 2016

Research sheds light on key genes essential to pathogenesis of Helicobacter pylori

Professor Frédéric Veyrier's most recent research, in collaboration with the team of Professor Hilde De Reuse at the Institut Pasteur, has shed light on key genes essential to the pathogenesis of Helicobacter pylori bacterium, which causes gastric infections.

21 Dec 2016

Novel approach combines imaging and electroanatomical mapping for targeted cardiac gene transfer

Gene therapy to repair damaged heart muscle is most likely to succeed if it can be injected at the site of ischemia where there is viable myocardium with reduced contractile ability, and a new technique that combines imaging and electroanatomical mapping does just that.

15 Nov 2016

Novel ‘gene therapy in a box’ could effectively deliver modified blood stem cells

A table-top device that enables medical staff to genetically manipulate a patient's blood to deliver potential new therapies for cancer, HIV and other diseases would eliminate the need for multi-million-dollar "clean rooms," making gene therapy more possible for even the poorest of countries.

24 Oct 2016

Antimicrobial copper touch surfaces may help fight global threat of antibiotic resistance

A key presentation at the Infection Control show within the Patient First conference on 22 November will look at a simple engineering solution to help fight the global threat of antibiotic resistance: antimicrobial copper touch surfaces.

21 Oct 2016

Clinical study results reveal long-term safety of gene transfer to treat macular degeneration

The long-term safety of gene transfer to treat neovascular age-related macular degeneration (NVAMD), and the production of two therapeutic proteins encoded by those genes for at least 2.5 years in the eyes of patients with advanced NVAMD are demonstrated in the results of a clinical trial published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers.

20 Oct 2016