Gene Therapy News
May 1, 2016

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More Gene Therapy News
May 1, 2016

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

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

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

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

Rare Respiratory Disease Gene Carriers Actually Have Increased Lung Function

Mar. 4, 2016 — New research has revealed the healthy carriers of a gene that causes a rare respiratory disease are taller and larger than average, with greater respiratory capacity. The disease, alpha1-antitrypsin ... read more

Normal Stem Cells Linked to Aggressive Prostate Cancer

Feb. 29, 2016 — A study that revealed new findings about prostate cells may point to future strategies for treating aggressive and therapy-resistant forms of prostate cancer, report ... read more

Feb. 24, 2016 — A new treatment for aplastic anemia is based on the transport of the telomerase gene to the bone marrow cells using gene therapy, a completely new strategy in the treatment of aplastic ... read more

Potential Treatment for Friedreich's Ataxia Identified

Feb. 16, 2016 — Researchers have identified synthetic RNA and DNA that reverses the protein deficiency causing Friedreich’s ataxia, a neurological disease for which there is currently no ... read more

Researchers Identify Way Radiation May Fight Cancer Cells Escaping Immune System

Feb. 1, 2016 — A team of researchers is fighting cancers using a combination of therapies and recently found ways that radiation could maximize responses to novel immune-based therapeutic approaches to fight ... read more

In Lung Cancer, Not All HER2 Alterations Are Created Equal

Jan. 28, 2016 — Study shows two distinct causes of HER2 activation in lung cancer: mutation of the gene and amplification of the gene. In patient samples of lung adenocarcinoma, 3 percent were found to have HER2 ... read more

Gene Therapy for Rare Bleeding Disorder Achieves Proof-of-Concept

Jan. 20, 2016 — Hematology researchers have used a single injection of gene therapy to correct a rare bleeding disorder, factor VII deficiency, in dogs. This success in large animals holds considerable potential for ... read more

Common Gene Mutation Bad for Liver Values, Good for Blood Lipids in Children

Jan. 13, 2016 — A common mutation in one gene raises liver values but at the same time improves blood lipid values in healthy children, according to a recent study. Children who carry the gene mutation had higher ... read more

Novel RNA Delivery System May Treat Incurable Blood Cancers

Jan. 5, 2016 — Mantle Cell Lymphoma is considered the most aggressive known blood cancer, and available therapies are scarce. A new study offers tangible hope of curing the currently incurable cancer -- and others ... read more

Three Hits to Fight Lung Cancer

Jan. 4, 2016 — Cancers with KRAS-related gene mutations might benefit from a triple therapy with two experimental drugs plus radiation therapy, a new study in mice has ... read more

Dec. 16, 2015 — Proteins are like bricks that form our cells and they are built by the orders given by our genetic material, DNA. In human diseases, eventually DNA alterations modify proteins and they don't do ... read more

Dec. 12, 2015 — Results from a long-term clinical trial conducted by cancer researchers show that combining radiation treatment with 'suicide gene therapy' provides a safe and effective one-two punch ... read more

Gene Therapy Used to Extend Estrogen's Protective Effects on Memory

Dec. 8, 2015 — The hormone estrogen helps protect memory and promote a healthy brain, but this effect wanes as women age, and even estrogen replacement therapy stops working in humans after age 65. Now researchers ... read more

Shrinking Tumors With an RNA Triple-Helix Hydrogel Glue

Dec. 7, 2015 — An efficient and effective delivery vehicle for gene therapy has been developed by researchers who have used it to shrink tumors by nearly 90 percent in a pre-clinical model of triple-negative breast ... read more

Characteristics That May Increase a Breast Cancer Survivor's Risk of Developing Leukemia Following Treatment Identified

Dec. 7, 2015 — A new analysis indicates that certain characteristics may increase a breast cancer survivor's risk of developing leukemia after undergoing chemotherapy and/or radiation. The findings are a first ... read more

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