Viral Vector News and Research

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Viral Vector is a type of virus used in cancer therapy. The virus is changed in the laboratory and cannot cause disease. Viral vectors may produce tumor antigens (proteins found on a tumor cell) to stimulate an antitumor immune response in the body. Viral vectors may also be used to carry genes that can change cancer cells back to normal cells.

Study finds that gene therapy can clip out genetic material associated with heart failure

Gene therapy can clip out genetic material linked to heart failure and replace it with the normal gene in human cardiac cells, according to a study led by researchers from the Cardiovascular Research Center at Icahn School of Medicine at Mount Sinai.

30 Apr 2015

UC Davis researchers settle long-standing controversy surrounding Canavan disease

UC Davis investigators have settled a long-standing controversy surrounding the molecular basis of an inherited disorder that historically affected Ashkenazi Jews from Eastern Europe but now also arises in other populations of Semitic descent, particularly families from Saudi Arabia.

28 Apr 2015

Novasep, Celladon sign agreement to develop and supply MYDICAR drug substance

Novasep, a leading supplier of services and technologies for the life sciences and chemical industries, and Celladon Corporation, a clinical-stage cardiovascular gene therapy company, today announce that they have signed a Development, Manufacturing and Supply Agreement pursuant to which, if supported by upcoming MYDICAR clinical data, Novasep would manufacture MYDICAR drug substance through 2018 with extension options through 2020.

26 Mar 2015

Study: New gene therapy safe, effective for patients with hemophilia B

A multi-year, ongoing study suggests that a new kind of gene therapy for hemophilia B could be safe and effective for human patients. Published in the journal Science Translational Medicine, the research showed that a reprogrammed retrovirus could successfully transfer new factor IX (clotting) genes into animals with hemophilia B to dramatically decrease spontaneous bleeding.

12 Mar 2015

Penn researcher named a recipient of 2015 Paul Ehrlich and Ludwig Darmstaedter Prize

University of Pennsylvania cancer and HIV expert Carl June, MD, has been named one of two recipients of the 2015 Paul Ehrlich and Ludwig Darmstaedter Prize for his outstanding work in cancer immunotherapy. Since 1952, the Paul Ehrlich and Ludwig Darmstaedter Prize has been awarded to scientists who have made great advancements in the fields in which Paul Ehrlich worked, in particular immunology, cancer research, microbiology, and chemotherapy.

12 Mar 2015

New strategy may ensure safety of adult epidermal stem cells before performing treatments

A team of European researchers has devised a strategy to ensure that adult epidermal stem cells are safe before they are used as treatments for patients. The approach involves a clonal strategy where stem cells are collected and cultivated, genetically modified and single cells isolated before being rigorously tested to make sure they meet the highest possible safety criteria.

28 Feb 2015

Study results pave way for new clinical trial for glioblastoma patients

Immune cells engineered to seek out and attack a type of deadly brain cancer were found to be both safe and effective at controlling tumor growth in mice that were treated with these modified cells, according to a study published in Science Translational Medicine by a team from the Perelman School of Medicine at the University of Pennsylvania and the Novartis Institutes for BioMedical Research.

19 Feb 2015

Novasep selected to commercially biomanufacture Celladon's Mydicar API

Novasep, a leading supplier of services and technologies for the life sciences industry, today announces that it has entered into an agreement with Celladon, a clinical-stage biotechnology company. Novasep will prepare to supply the drug substance for MYDICAR®.

19 Feb 2015

New gene therapy for Acute Intermittent Porphyria

Angeles suffers from a severe and rare genetic disease called Acute Intermittent Porphyria (AIP). This means, one of her genes restrains her liver to produce a specific protein needed for the metabolism of the blood. During an AIP attack Angeles usually feels intense abdominal pain, and her mental status changes. Women live at an increased risk to trigger the symptoms, because porphyria is related to the menstrual cycle.

2 Jul 2014

Cancer drug shows promise in treating blood cell disorders

Scientists working to make gene therapy a reality have solved a major hurdle: how to bypass a blood stem cell's natural defenses and efficiently insert disease-fighting genes into the cell's genome.

27 Jun 2014

Researchers reproduce tumor chromosomal translocations in human cells

Scientists from the Spanish National Cancer Research Centre and the Spanish National Cardiovascular Research Centre have been able to reproduce, for the first time in human cells, chromosomal translocations associated with two types of cancer: acute myeloid leukaemia and Ewing's sarcoma.

4 Jun 2014

Study: Sufficient copies of SMN1 gene extend survival in animals with spinal muscular atrophy

To make up for insufficient amounts of SMN protein, the cause of the inherited neuromuscular disease spinal muscular atrophy (SMA), researchers have successfully delivered a replacement SMN1 gene directly to the spinal cords of animal models of SMA.

23 May 2014

Inserm plays key role in developing European research

The French National Institute of Health and Medical Research (Inserm) is currently the leading biomedical research organisation in Europe, and plays a key role in developing European research. To mark its 50th anniversary, Inserm wanted to assess the perceptions of Europeans regarding biomedical research.

10 Apr 2014

Cedars-Sinai earns grant to conduct clinical trial of gene therapy product for patients with Lou Gehrig's disease

The Cedars-Sinai Regenerative Medicine Institute has received a $2.5 million grant from the Department of Defense to conduct animal studies that, if successful, could provide the basis for a clinical trial of a gene therapy product for patients with Lou Gehrig's disease, also called amyotrophic lateral sclerosis, or ALS.

2 Apr 2014

Transgene raises €65.5M via two-step capital increase

Transgene SA, a French biopharmaceutical company focused on discovering, developing and manufacturing targeted immunotherapies for the treatment of cancer and infectious diseases, announces that it has raised a total of €65.5 million via a two-step capital increase.

25 Mar 2014

Researchers identify new player in onset and progression of heart failure

A team of cardiovascular researchers from the Cardiovascular Research Center at Icahn School of Medicine at Mount Sinai, Sanford-Burnham Medical Research Institute, and University of California, San Diego have identified a small, but powerful, new player in the onset and progression of heart failure.

13 Mar 2014

Horizon Discovery, Sirion Biotech ink supply and distribution agreement

Horizon Discovery, a leading provider of research tools to support translational genomics and the development of personalized medicines, today announced it has signed a supply and distribution agreement with Sirion Biotech GmbH.

17 Feb 2014

New ways to treat substance abuse, neurological diseases and mental illnesses

Researchers at the University at Buffalo have found a way to change alcohol drinking behavior in rodents, using the emerging technique of optogenetics, which uses light to stimulate neurons.

7 Jan 2014

Researchers report new gene delivery method that targets tumor blood vessels

Working in mice, researchers at Washington University School of Medicine in St. Louis report developing a gene delivery method long sought in the field of gene therapy: a deactivated virus carrying a gene of interest that can be injected into the bloodstream and make its way to the right cells.

24 Dec 2013

New form of gene therapy shows promising outcomes for "bubble boy" disease

Researchers reported promising outcomes data for the first group of boys with X-linked severe combined immunodeficiency syndrome (SCID-X1), a fatal genetic immunodeficiency also known as "bubble boy" disease, who were treated as part of an international clinical study of a new form of gene therapy. The mechanism used to deliver the gene therapy is designed to prevent the serious complication of leukemia that arose a decade ago in a similar trial in Europe, when one-quarter of boys treated developed the blood cancer.

9 Dec 2013