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Human Gene Therapy Remove


July 2016
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Human Gene Therapy Press Releases

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12561085
By Mary Ann Liebert, Inc. publishers
Liver cancer can be triggered by mutations in cancer driver genes resulting from the insertion of adeno-associated virus (AAV) vectors used to deliver therapeutic genes, although this tumor-inducing role of AAV remains highly controversial.
12551599
By Mary Ann Liebert, Inc. publishers
A new study to understand why viral particles tend to accumulate in a specific location around a cell's nucleus in the first several hours after viral infection has shown this phenomenon to be a novel defense mechanism used by cells to block nuclear...
12546478
By Mary Ann Liebert, Inc. publishers
A new study in patients with the inherited metabolic disorder methylmalonic academia (MMA) found lower than expected levels of antibodies against the adeno-associated viral (AAV) vectors being developed for gene therapy to replace the enzyme (MUT) that...
12352106
By Mary Ann Liebert, Inc., publishers
Contact: Kathryn Ruehle, Mary Ann Liebert, Inc., (914) 740-2100, kruehle@liebertpub.com Malcolm K. Brenner, MD, PhD Receives Pioneer Award for Advances in Gene-Modified T Cells Targeting Cancer New Rochelle, NY—Malcolm K.
12343486
By Mary Ann Liebert, Inc., publishers
for Contributions to Efficient Vector Integration in Human Gene Therapy
12326568
By Mary Ann Liebert, Inc., publishers
A targeted gene silencing strategy blocks production of the dysfunctional huntingtin (Htt) protein, the cause of Huntington's disease, a fatal, inherited neurodegenerative disorder.
12317188
By Mary Ann Liebert, Inc., publishers
Contact: Vicki Cohn, Mary Ann Liebert, Inc, (914) 740-2100, ext. 2156, vcohn@liebertpub.com James Wilson, MD, PhD Receives Pioneer Award for Research on Gene Therapy and AAV Gene Delivery Systems New Rochelle, NY—James M.
12299634
By Mary Ann Liebert, Inc., publishers
Marina Cavazzana, MD, PhD, Paris Descartes University, and Adrian J. Thrasher, MD, PhD, University College London Institute of Child Health, have been honored with the Pioneer Award for basic and clinical gene therapy for immunodeficiency disorders.
12247355
By Mary Ann Liebert, Inc., publishers
Selected by Blue Ribbon Panel; Essays to be Published in Human Gene Therapy
12223489
By Mary Ann Liebert, Inc., publishers
Contact: Vicki Cohn, Mary Ann Liebert, Inc, (914) 740-2100, ext.
12059856
By Mary Ann Liebert, Inc., publishers
A novel therapeutic approach called exon skipping involves bypassing a disease-causing mutation in a gene to restore normal gene expression and protein production.
11998193
By Mary Ann Liebert, Inc., publishers
Human Gene Therapy is expanding its editorial coverage with the launch of Human Gene Therapy Clinical Development early next year.

All Press Releases

By Mary Ann Liebert, Inc. publishers
The encouraging results of early stage clinical studies and the tremendous amount of preclinical data demonstrating the feasibility and promise of gene therapy to treat disorders of the central nervous system (CNS) are driving new advances for the...
By Mary Ann Liebert, Inc. publishers
Diseases of the eye that cause vision loss and blindness, especially neurodegenerative disorders affecting the retina, are ideal targets for gene therapy, including gene replacement and promising corrective gene editing strategies.
12561085
By Mary Ann Liebert, Inc. publishers
Liver cancer can be triggered by mutations in cancer driver genes resulting from the insertion of adeno-associated virus (AAV) vectors used to deliver therapeutic genes, although this tumor-inducing role of AAV remains highly controversial.
By Mary Ann Liebert, Inc. publishers
A new study shows that the anti-tumor effect of oncolytic virus therapy is significantly greater in mice when the virus is genetically modified to express a junction opening (JO) protein, which helps the cancer-killing agent better penetrate solid...
By Mary Ann Liebert, Inc. publishers
Genetic regulation of the various types of blood cells in zebrafish and humans is highly similar, making it relatively easy and cost-effective to perform genetic, chemical, imaging and other molecular studies on this invaluable model organism to study...
By Mary Ann Liebert, Inc. publishers
Promising results from the first clinical trials of globin gene transfer to treat beta-thalassemias-inherited forms of anemia-have eliminated the need for blood transfusions in some individuals.
12551599
By Mary Ann Liebert, Inc. publishers
A new study to understand why viral particles tend to accumulate in a specific location around a cell's nucleus in the first several hours after viral infection has shown this phenomenon to be a novel defense mechanism used by cells to block nuclear...
12546478
By Mary Ann Liebert, Inc. publishers
A new study in patients with the inherited metabolic disorder methylmalonic academia (MMA) found lower than expected levels of antibodies against the adeno-associated viral (AAV) vectors being developed for gene therapy to replace the enzyme (MUT) that...
By Mary Ann Liebert Inc., publishers
X-linked severe combined immunodeficiency (SCID-X1) was the test-case for demonstrating the feasibility of using gene therapy targeting hematopoietic stem cells to reconstitute the human immune system and provide effective long-term treatment for a...
By Mary Ann Liebert Inc., publishers
A novel HIV-based lentiviral vector can introduce a gene to pancreatic tumor cells that makes them more sensitive to the chemotherapeutic drug gemcitabine, without integrating into cellular DNA.
By Mary Ann Liebert Inc., publishers
New initiatives by the National Center for Advancing Translational Sciences (NCATS) to use gene therapy approaches to treat rare diseases and especially promising aspects of gene transfer and gene editing technology, such as adeno-associated viral (AAV)..
By Mary Ann Liebert Inc., publishers
Therapeutic gene transfer to specific brain regions in animal models of Alzheimer's disease has uncovered multiple potential treatment approaches that deserve to be considered as candidates for clinical applications, according to the article “Gene...
By Mary Ann Liebert Inc., publishers
The conclusion drawn from a recent study that insertion of adeno-associated virus 2 (AAV2) into human DNA causes mutations leading to the development of hepatocellular carcinoma (HCC) was resoundingly rejected by leading researchers in the fields of gene.
By Mary Ann Liebert Inc., publishers
Mitchell Finer, PhD, a scientist with expertise in biochemistry and molecular biology, who has had an integral role in the success of numerous biotechnology companies, including most recently as chief scientific officer of gene therapy innovator bluebird.
By Mary Ann Liebert Inc., publishers
Highly anticipated phase III clinical trial results of Spark Therapeutics's gene therapy to treat visual impairment are due by year-end and could have profound implications for the broader gene therapy field, leading to the first approved product in the..
By Mary Ann Liebert Inc., publishers
A gene therapy approach to treating the progressive muscle wasting disorder Duchenne muscular dystrophy (DMD) that does not replace the mutated DMD gene but instead delivers the gene for ITGA7, a protein in skeletal muscle, led to reduced symptoms and...
By Mary Ann Liebert Inc., publishers
Mice lacking the protein retGC1, which is deficient in humans suffering Leber congenital amaurosis-1 (LCA1), a disorder that causes severe visual impairment beginning in infancy, received gene therapy to replace retGC1 and showed fully restored visual...
By Mary Ann Liebert Inc., publishers
Chimeric antigen receptor (CAR) T cells, which can specifically recognize and target tumor cells, have resulted in complete responses in patients with leukemia, and although equally promising for treating lymphoma, obstacles remain and individual patient.
By Mary Ann Liebert Inc., publishers
CRISPR/Cas systems for genome editing have revolutionized biological research over the past three years, and their ability to make targeted changes in DNA sequences in living cells with relative ease and affordability is now being applied to clinical...
By Mary Ann Liebert Inc., publishers
To overcome the off-target mutations that commonly occur with CRISPR/Cas9 genome editing methods, researchers at Harvard Medical School and Massachusetts General Hospital have developed two strategies that greatly improve the specificity of RNA-guided...
By Mary Ann Liebert Inc., publishers
Present New Initiative on Ethics of Human Gene Editing Technology
By Mary Ann Liebert Inc., publishers
Recent evidence demonstrating the feasibility of using novel CRISPR/Cas9 gene editing technology to make targeted changes in the DNA of human embryos  is forcing researchers, clinicians, and ethicists to revisit the highly controversial issue of...
By Mary Ann Liebert, Inc., publishers
Mary Ann Liebert, Inc., publishers is pleased to announce the appointment of Terry R.
By Mary Ann Liebert, Inc., publishers
Recognized for their pioneering work in the development of  gene transfer technology using retroviral vectors to deliver therapeutic genes into cells, Richard C.
By Mary Ann Liebert, Inc., publishers
Recognized for her pioneering work to develop gene therapy for hemophilia spanning several decades, taking it from the laboratory into human clinical trials, is Katherine A. High, MD, Spark Therapeutics.
By Mary Ann Liebert, Inc., publishers
Zelig Eshhar, PhD, The Weizmann Institute of Science and Sourasky Medical Center, and Carl H.
By Mary Ann Liebert, Inc., publishers
Excessive and often lethal blood levels of bilirubin can result from mutations in a single gene that are the cause of the metabolic disease known as Crigler-Najjar syndrome type 1 (CNS1).
12352106
By Mary Ann Liebert, Inc., publishers
Contact: Kathryn Ruehle, Mary Ann Liebert, Inc., (914) 740-2100, kruehle@liebertpub.com Malcolm K. Brenner, MD, PhD Receives Pioneer Award for Advances in Gene-Modified T Cells Targeting Cancer New Rochelle, NY—Malcolm K.
12343486
By Mary Ann Liebert, Inc., publishers
for Contributions to Efficient Vector Integration in Human Gene Therapy
By Mary Ann Liebert, Inc., publishers
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.

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