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.

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

Alliance for Cancer Gene Therapy grants nearly $25M to conduct cancer related gene therapy research

As the potential of gene and cell therapy in fighting cancers grows and oncologists across the nation explore the burgeoning science, doctors have uncovered a stunning development – cancers can be treated using patients' own cells as a weapon, and can eliminate the harrowing, often debilitating effects of chemotherapy, radiation and surgery.

28 Dec 2013

Stem cell-based gene therapy shows long-term outcomes for treatment of genetic skin diseases

Stem cell-based gene therapy holds promise for the treatment of devastating genetic skin diseases, but the long-term clinical outcomes of this approach have been unclear.

27 Dec 2013

Cell transplantation, gene therapy combination to open new therapeutic avenues for Alzheimer's disease

In a recent study published in the Neural Regeneration Research (Vol. 8, No. 33, 2013), Prof. Feng Li and team from Zhongshan School of Medicine, Sun Yat-sen University in China, synthesized a 19-nt oligonucleotide targeting BACE1, the key enzyme in amyloid beta protein (Aβ) production, and introduced it into the pSilenCircle vector to construct a short hairpin (shRNA) expression plasmid against the BACE1 gene.

24 Dec 2013

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

Finding suggests new route for packaging RNA molecules of interest into nanovesicles

The role of microRNAs is fundamental for the correct moment-to-moment adjustment in the expression of target genes. "Before this study, we already knew that these small molecules could be packaged into small vesicles and exported to the extracellular space, to be later captured by other cells and in this way play an important role in intercellular communication," explains CNIC researcher Carolina Villarroya, the first author on the study.

23 Dec 2013

Engineered immune cells show antitumor activity in two patients with advanced cancers

Engineered immune cells, called CARTmeso cells, designed to direct antitumor immune responses toward tumors that carry a protein called mesothelin, showed antitumor activity in two patients with advanced cancers that had not responded to prior treatments, according to a study published in Cancer Immunology Research, a journal of the American Association for Cancer Research.

22 Dec 2013

UCLA scientists receive $21M grant to conduct groundbreaking clinical trials on stem cell science

Scientists from UCLA are now bringing their groundbreaking stem cell science directly to patients in two exciting new clinical trials scheduled to begin in early 2014, thanks to funding from California's stem cell agency.

17 Dec 2013

Moderate drinking may bolster our immune system and help fight off infection: Study

Medical science has known for years that people who drink moderate amounts of alcohol actually have a reduced risk of death. In general, they are healthier and have better cardiovascular function that those who don't drink alcohol at all.

17 Dec 2013

CHOP scientist receives 2013 E. Donnall Thomas Prize for scientific, clinical research in hemophilia

Katherine A. High, M.D., of The Children's Hospital of Philadelphia (CHOP) was honored for her trailblazing scientific and clinical research in the bleeding disorder hemophilia when she received the 2013 E. Donnall Thomas Prize from the American Society of Hematology (ASH) at its annual meeting on Dec. 9 in New Orleans.

15 Dec 2013

CHOP scientist receives 2013 E. Donnall Thomas Prize for research in bleeding disorder hemophilia

Katherine A. High, M.D., of The Children's Hospital of Philadelphia was honored for her trailblazing scientific and clinical research in the bleeding disorder hemophilia when she received the 2013 E. Donnall Thomas Prize from the American Society of Hematology at its annual meeting on Dec. 9 in New Orleans.

14 Dec 2013

Psychiatrists increasingly refuse to accept private insurance as payment

Access to mental health care has become a prominent issue in Congress following mass shootings around the country. But a new study in JAMA Psychiatry, led by researchers at Weill Cornell Medical College, suggests that unless those in need of help have deep pockets, they might have a hard time finding a psychiatrist that will provide the needed services.

13 Dec 2013

Scientists bring stem cell science directly to patients

Scientists from UCLA's Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research are bringing stem cell science funded by the California Institute of Regenerative Medicine (CIRM), the state stem cell agency, directly to patients in two exciting new clinical trials scheduled to begin in early 2014.

13 Dec 2013

Researchers discover new therapeutic approach for hemophilia

Researchers at the UNC School of Medicine and the Medical College of Wisconsin found that a new kind of gene therapy led to a dramatic decline in bleeding events in dogs with naturally occurring hemophilia A, a serious and costly bleeding condition that affects about 50,000 people in the United States and millions more around the world.

12 Dec 2013

New reports demonstrate the effectiveness of modified T cells in treating blood-borne cancers

At the 2013 American Society of Hematology meeting in Dec. 2013, James Kochenderfer, M.D., investigator in the Experimental Transplantation and Immunology Branch, NCI, presented findings from two clinical trials evaluating the use of genetically modified immune system T cells as cancer therapy.

12 Dec 2013

Understanding long-term clinical stability in hemophilia patients living with HIV

People with the genetic blood clotting disorder hemophilia who have been infected with HIV for decades have an increased proportion of immune cells in their blood that specifically target HIV. This protective immune response helps chronically infected hemophilia patients survive, even during periods of HIV activity, according to a study published in BioResearch Open Access, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers.

12 Dec 2013

Scientists explore mechanisms of viruses' shells

Viruses are like small vessels containing an active component, the genetic material, that can infect a host cell. The vessel, called capsid or vector, is basically a shell that changes its shape when it penetrates a cell to infect it, and may even break into pieces.

12 Dec 2013

New drug delivery system successfully treats advanced-stage ovarian cancer in mice

Scientists at Rutgers University have developed a targeted drug delivery system that they believe could make ovarian cancer more treatable and increase survival rates for the most deadly gynecological cancer in the United States.

9 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

Genetically engineered cell therapies demonstrate early efficacy, safety in patients with blood disorders

A series of advancements in genetically engineered cell therapies demonstrate early efficacy and safety in patients with blood disorders for whom standard treatments have been unsuccessful, according to data showcased today during the 55th American Society of Hematology (ASH) Annual Meeting and Exposition in New Orleans.

9 Dec 2013

Gene Therapy News and Research

Further Reading

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

Alliance for Cancer Gene Therapy grants nearly $25M to conduct cancer related gene therapy research

Stem cell-based gene therapy shows long-term outcomes for treatment of genetic skin diseases

Cell transplantation, gene therapy combination to open new therapeutic avenues for Alzheimer's disease

Researchers report new gene delivery method that targets tumor blood vessels

Finding suggests new route for packaging RNA molecules of interest into nanovesicles

Engineered immune cells show antitumor activity in two patients with advanced cancers

UCLA scientists receive $21M grant to conduct groundbreaking clinical trials on stem cell science

Moderate drinking may bolster our immune system and help fight off infection: Study

CHOP scientist receives 2013 E. Donnall Thomas Prize for scientific, clinical research in hemophilia

CHOP scientist receives 2013 E. Donnall Thomas Prize for research in bleeding disorder hemophilia

Psychiatrists increasingly refuse to accept private insurance as payment

Scientists bring stem cell science directly to patients

Researchers discover new therapeutic approach for hemophilia

New reports demonstrate the effectiveness of modified T cells in treating blood-borne cancers

Understanding long-term clinical stability in hemophilia patients living with HIV

Scientists explore mechanisms of viruses' shells

New drug delivery system successfully treats advanced-stage ovarian cancer in mice

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

Genetically engineered cell therapies demonstrate early efficacy, safety in patients with blood disorders

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