Gene Therapy News
September 5, 2016

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updated 12:33pm EDT

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September 5, 2016

Collisions During DNA Replication, Transcription Contribute to Mutagenesis

June 29, 2016 — Replication-transcription head-on collisions contribute to mutagenesis, report ... read more

Who Should Pay for Gene Therapy?

May 26, 2016 — As gene therapy trials show promise to cure or ameliorate several diseases, it is time to explore ways to pay for the treatments, according to a new commentary. Noting the potential of gene therapy ... read more

Novel Gene Therapy Shows Potential for Lung Repair in Asthma

May 18, 2016 — A new study has demonstrated a way to deliver a nanoparticle-based gene therapy, in order to repair lungs damaged by chronic allergic asthma and to reduce inflammation that causes asthma attacks. The ... read more

Validation of Screening Tool for ROS1 Gene Rearrangements

May 11, 2016 — Immunohistochemistry (IHC) is an effective tool that can be used for identifying proto-oncogene 1 receptor tyrosine kinase (ROS1) gene rearrangements and screening patients for the administration of ... read more

Gene Replacement Therapy Offers Viable Treatment Option for Fatal Disease

May 5, 2016 — Spinal muscular atrophy (SMA) is a disease that causes progressive degeneration in the nerve cells that control muscles, thereby causing muscle weakness and eventually death. SMA affects ... read more

Gene Therapy Halts Pulmonary Hypertension Progression in Large Animal Pre-Clinical Study

Apr. 28, 2016 — Scientists have used a novel gene therapy to halt the progression of pulmonary hypertension, a form of high blood pressure in the lung blood vessels that is linked to heart ... read more

Gene Therapy Shows Long-Term Benefit for Treating Rare Blindness

Apr. 28, 2016 — Pioneering gene therapy has restored some vision to patients with a rare form of genetic blindness for as long as four years, raising hopes it could be used to cure common causes of vision loss, new ... read more

Altered Primary Chromatin Structures: Implications in Cancer Development

Apr. 25, 2016 — Alterations in the primary structure of chromatin resulting from imprecise arrangements of nucleosomes along DNA has been the focus of recent research, and in particular, its functional implications ... read more

Study Finds Explanation for Some Treatment-Resistant Breast Cancers

Apr. 21, 2016 — Investigators have identified gene alterations that may explain why triple-negative breast cancer is resistant to most existing treatments, and suggests that a targeted therapy currently in clinical ... read more

Gene Therapy Shows Early Success Against 'Bubble Boy' Disease

Apr. 20, 2016 — Early results suggest novel gene therapy is safe and effective for treatment of inherited immune disease, according to scientists. The study involved five males with X-linked severe combined ... read more

New Study Shows Children Benefited Most from Gene Therapy for LCA, a Rare Eye Disease

Apr. 15, 2016 — Scientists have completed a two-year Phase I clinical trial which showed that children showed the greatest benefit from gene therapy for treatment of Leber congenital amaurosis or severe early ... read more

Specific Gene in the Tumor Determines the Effectiveness of Cancer Treatment

Apr. 14, 2016 — A cancer treatment can be basically effective but, equally, it may have negative consequences. Therefore, it has not been possible to determine prior to treatment whether a patient will benefit from ... read more

Novel Mechanism of Crizotinib Resistance in a ROS1+ NSCLC Patient

Apr. 11, 2016 — Molecular analysis of a tumor biopsy from a proto-oncogene 1 receptor tyrosine kinase positive (ROS1+) patient with acquired crizotinib resistance revealed a novel mutation in the v-kit Hardy ... read more

New Tool Enables Scientists to Interpret 'Dark Matter' DNA

Apr. 4, 2016 — Scientists have invented a new way to read and interpret the human genome. The computational method, called TargetFinder, can predict where non-coding DNA -- the DNA that does not code for proteins ... read more

Gene Transfer Shows Promise for Treating Heart Failure

Mar. 30, 2016 — Use of intracoronary gene transfer among heart failure patients resulted in increased left ventricular function beyond standard heart failure therapy, according to a ... read more

Mar. 24, 2016 — Two new gene modification methods have been developed: lsODN (long single-stranded oligodeoxynucleotide) and 2H2OP (two-hit two-oligo with plasmid). These methods use CRISPR (Clustered Regularly ... read more

Mar. 17, 2016 — There exist several oncogenes that drive cancer. In many cases, however, the oncogenes themselves are not sufficient and must be complemented with other mutations before cancer develops. Researchers ... read more

Mar. 10, 2016 — Neurons in the brain that have been supplemented with a synthetic gene can be remotely manipulated by a magnetic field, scientists have shown. The finding has implications for possible future ... read more

Modified Form of CRISPR Acts as a Toggle Switch to Control Gene Expression in Stem Cells

Mar. 10, 2016 — Combining the two most powerful biological tools of the 21st century, scientists have modified how the genome of induced pluripotent stem cells (iPSCs) is read for the first time using a variation of ... read more

Scientists Use Synthetic Gene and Magnets to Alter Behavior of Mice, Fish

Mar. 7, 2016 — Scientists have demonstrated that neurons in the brain that have been supplemented with a synthetic gene can be remotely manipulated by a magnetic field. This has implications for future treatment of ... read more

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