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.

Researchers map two gene mutations that trigger retinal disease leading to macular degeneration

Two gene mutations that trigger a retinal disease that causes blindness in one in 5,000 males have been mapped, leading to the potential for new therapeutic treatments.

7 Nov 2016

WSU researcher finds a way to reduce development of cancer cells

A Washington State University researcher has developed a way to reduce the development of cancer cells that are an infrequent but dangerous byproduct of gene therapy.

5 Nov 2016

Takara Bio to provide SMART-Seq v4 Ultra Low Input RNA Kit for Sequencing to Allen Institute

Takara Bio USA, Inc. announced today that its SMART-Seq v4 Ultra Low Input RNA Kit for Sequencing has been selected for the development of gene expression profiles of individual brain cells as part of the Allen Cell Types Database, a public resource available from the Allen Institute for Brain Science.

2 Nov 2016

Heart problems from common cancer treatment can be alleviated by using gene therapy

Researchers of the Wihuri Research Institute and the University of Helsinki, Finland, have found that some of the harmful effects of a commonly used cancer drug can be alleviated by using gene therapy that stimulates blood vessel growth in the heart.

31 Oct 2016

Gene therapy may be effective method for treating Niemann-Pick disease, type C1

For the first time, National Institutes of Health researchers have demonstrated in mice that gene therapy may be the best method for correcting the single faulty gene that causes Niemann-Pick disease, type C1 (NPC1).

26 Oct 2016

Researchers find link between estrogen levels in lung tissue and SMLA in postmenopausal women

Thanks to advances in medical imaging, the detection rate for synchronous multiple lung adenocarcinoma (SMLA) has been on the rise. Cases of SMLA in Japanese women have been on the rise despite having a national smoking rate of less than 10% in recent years.

26 Oct 2016

Yale researchers use new gene editing technique to correct mutations that cause thalassemia

A Yale-led research team used a new gene editing strategy to correct mutations that cause thalassemia, a form of anemia.

26 Oct 2016

Novel ‘gene therapy in a box’ could effectively deliver modified blood stem cells

A table-top device that enables medical staff to genetically manipulate a patient's blood to deliver potential new therapies for cancer, HIV and other diseases would eliminate the need for multi-million-dollar "clean rooms," making gene therapy more possible for even the poorest of countries.

24 Oct 2016

Cedars-Sinai receives FDA approval to examine safety of combination stem cell-gene therapy in ALS patients

Cedars-Sinai regenerative medicine investigators have received approval from the U.S. Food and Drug Administration to test a combination stem cell-gene therapy they developed to stall the progression of amyotrophic lateral sclerosis, a neurological disease that causes progressive paralysis and ultimately death.

21 Oct 2016

Clinical study results reveal long-term safety of gene transfer to treat macular degeneration

The long-term safety of gene transfer to treat neovascular age-related macular degeneration (NVAMD), and the production of two therapeutic proteins encoded by those genes for at least 2.5 years in the eyes of patients with advanced NVAMD are demonstrated in the results of a clinical trial published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers.

20 Oct 2016

Domain Therapeutics and Medicxi partner to launch Mavalon Therapeutics

Domain Therapeutics, a biopharmaceutical company that specializes in the discovery and development of G protein-coupled receptor (GPCR) drugs, today announces the creation of Mavalon Therapeutics, a company focused on stopping the progression of Parkinson’s disease.

19 Oct 2016

Biomedical engineers harvest bacterial genes that can improve electrical signaling in human cells

Duke University biomedical engineers have harvested genes for ion channels from bacteria that, with a few tweaks, can create and enhance electrical signaling in human cells, making the cells more electrically excitable.

19 Oct 2016

Long-term clinical data, experience supports effectiveness of PRRT for treating neuroendocrine tumors

More than ten years of published clinical data and personal experience using PRRT-based targeted therapy of neuroendocrine tumors supports the effectiveness of this novel treatment approach and the ability to minimize and manage potential toxic side effects.

17 Oct 2016

New research reveals key protein involved in heart failure

A key protein that causes heart failure has been revealed through new research from a collaboration based in Kumamoto University, Japan.

13 Oct 2016

Genome engineering-based methods pave way for new treatment of patients with sickle cell disease

A team of physicians and laboratory scientists has taken a key step toward a cure for sickle cell disease, using CRISPR-Cas9 gene editing to fix the mutated gene responsible for the disease in stem cells from the blood of affected patients.

12 Oct 2016

Researchers explain why explain why some tumors recur after immunotherapy

Immunotherapy is a new and highly promising form of treatment for cancer. In many patients, however, tumors recur after immunotherapy.

7 Oct 2016

Study offers promise for therapeutic management of fetal and congenital genetic diseases in utero

A breakthrough study by research teams at Rosalind Franklin University of Medicine and Science and Oregon Health & Science University offers promise for therapeutic management of congenital diseases in utero using designer nucleotide sequences that can simply be injected into the fluid surrounding the developing fetus to potentially treat disabling-to-lethal genetic defects.

7 Oct 2016

Gene therapy for LPLD patients linked to lower frequency and severity of pancreatitis

Up to 6 years after receiving a single treatment with the gene therapy product lipoprotein lipase (LPL), patients with the debilitating genetic disease LPL deficiency (LPLD) had about 50% fewer episodes of pancreatitis than before receiving the treatment.

28 Sep 2016

BREXIT vote threatens to disrupt advances in gene and cell therapies in the UK

The recent BREXIT vote in the UK threatens to disrupt scientific progress and clinical advances in the development of innovative gene and cell therapies.

28 Sep 2016

Research shows degenerative diseases affecting the retina may be treatable with gene therapy

Researchers have demonstrated the ability to deliver a fully functional copy of the CLN3 gene to stem cells of patients with juvenile NCL, an inherited neurodegenerative disease in which a mutation in the CLN3 gene causes early-onset severe central vision loss.

26 Sep 2016

Gene Therapy News and Research

Further Reading

Researchers map two gene mutations that trigger retinal disease leading to macular degeneration

WSU researcher finds a way to reduce development of cancer cells

Takara Bio to provide SMART-Seq v4 Ultra Low Input RNA Kit for Sequencing to Allen Institute

Heart problems from common cancer treatment can be alleviated by using gene therapy

Gene therapy may be effective method for treating Niemann-Pick disease, type C1

Researchers find link between estrogen levels in lung tissue and SMLA in postmenopausal women

Yale researchers use new gene editing technique to correct mutations that cause thalassemia

Novel ‘gene therapy in a box’ could effectively deliver modified blood stem cells

Cedars-Sinai receives FDA approval to examine safety of combination stem cell-gene therapy in ALS patients

Clinical study results reveal long-term safety of gene transfer to treat macular degeneration

Domain Therapeutics and Medicxi partner to launch Mavalon Therapeutics

Biomedical engineers harvest bacterial genes that can improve electrical signaling in human cells

Long-term clinical data, experience supports effectiveness of PRRT for treating neuroendocrine tumors

New research reveals key protein involved in heart failure

Genome engineering-based methods pave way for new treatment of patients with sickle cell disease

Researchers explain why explain why some tumors recur after immunotherapy

Study offers promise for therapeutic management of fetal and congenital genetic diseases in utero

Gene therapy for LPLD patients linked to lower frequency and severity of pancreatitis

BREXIT vote threatens to disrupt advances in gene and cell therapies in the UK

Research shows degenerative diseases affecting the retina may be treatable with gene therapy

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