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 report highlights serious health problems faced by spinal cord injury patients

Paralysis is just one of the many serious health problems faced by patients who suffer spinal cord injuries.

16 Feb 2017

Gene therapy treats myotubular myopathy in dogs

The rare disorder, called myotubular myopathy, or MTM, affects only males. It causes fatal muscle wasting. Both dogs and boys with the disease typically succumb in early life due to breathing difficulties.

16 Feb 2017

Signals from fat cells may aid in diagnostics for metabolic conditions

Fat cells are not simply big blobs of lipid quietly standingby in the body--instead, they send out hormones and other signaling proteins that affect many types of tissues.

15 Feb 2017

Penn Medicine's ODC collaborates with FAST to develop gene therapy for Angelman syndrome

Penn Medicine's Orphan Disease Center announces a new partnership with FAST to study gene therapy approaches to treat Angelman syndrome.

14 Feb 2017

Scientists identify how imbalance of calcium in mitochondria may contribute to Alzheimer's disease

Calcium in the mitochondria -- the energy factory of cells -- may be one of the keys to understanding and treating Alzheimer's disease and dementia.

14 Feb 2017

Review shows lipid nanoparticles as highly promising delivery systems in gene therapy

Lipid nanoparticles (SLNs and NLCs) are regarded as highly promising systems for delivering nucleic acids in gene therapy.

14 Feb 2017

Northwestern Medicine scientists discover retinal cell that may cause myopia

Northwestern Medicine scientists have discovered a cell in the retina that may cause myopia when it dysfunctions. The dysfunction may be linked to the amount of time a child spends indoors and away from natural light.

6 Feb 2017

Research unravels molecular mechanisms of antisense oligonucleotides

Stanley T. Crooke, MD, PhD, CEO of Ionis Pharmaceuticals and recipient of the 2016 Lifetime Achievement Award from the Oligonucleotide Therapeutic Society presents a detailed look at the mechanisms that underlie antisense drug activity in the article entitled "Molecular Mechanisms of Antisense Oligonucleotides," published in Nucleic Acid Therapeutics, a peer-reviewed journal from Mary Ann Liebert, Inc. publishers.

2 Feb 2017

Study reveals new molecular mechanism vital for associative memory encoding in the hippocampus

A new study, published on the cover of the scientific journal Biological Psychiatry and lead by Dr Carlos Saura from the Institut de Neurociències at the Universitat Autònoma de Barcelona, reveals a new molecular mechanism essential for associative memory encoding in the hippocampus.

31 Jan 2017

Scientists restore partial hearing and balance in deaf mice using new gene therapy

Using a novel form of gene therapy, scientists from Harvard Medical School and the Massachusetts General Hospital have managed to restore partial hearing and balance in mice born with a genetic condition that affects both.

28 Jan 2017

New MIP system for therapeutic gene delivery can enhance transgene expression using AAV vector

Advanced engineering of a mini-intronic plasmid (MIP) system designed to carry a therapeutic gene can significantly enhance the expression of the transgene delivered using an adeno-associated viral (AAV) vector.

28 Jan 2017

New gene therapy for Pompe disease may replace currently available treatments

After decades investigating a rare, life-threatening condition that cripples the muscles, Duke Health researchers have developed a gene therapy they hope could enhance or even replace the only FDA-approved treatment currently available to patients.

27 Jan 2017

Novel gene therapy restores partial hearing and balance in mice born with genetic deafness

27 Jan 2017

Salk Institute researchers reveal how to curb immune enthusiasm

Normally when we think of viruses, from the common cold to HIV, we want to boost people's immunity to fight them.

20 Jan 2017

MedCision announce participation at upcoming conference on cell and gene therapy

MedCision, a leader in the automation of pre-clinical and clinical processes, today announced the company’s participation in Phacilitate Cell and Gene Therapy 2017, a conference with the most comprehensive cell and gene therapy agenda in the industry.

13 Jan 2017

Orphan Disease Center establishes new initiative that focuses on ALS

The Orphan Disease Center in the Perelman School of Medicine at the University of Pennsylvania has established a new Program of Excellence for Motor Neuron Disease.

10 Jan 2017

New research suggests novel combination approach to fight against gliomas

"Devastating" and "dismal." That's how leading researchers describe the present outlook for malignant brain tumors. The median survival rate for patients with glioblastoma multiforme, or GBM, is a mere 14.2 months.

4 Jan 2017

AAV-based gene therapy to treat liver disorders advances into human testing

Liver-directed gene therapy delivered using adeno-associated viral (AAV) vectors to treat diseases such as hemophilia have advanced into human testing.

30 Dec 2016

Studies in non-human primates provide more predictive results in genetic causes of blindness

Many gene therapy-based approaches are in development to combat genetic and other causes of blindness and vision loss, and much can be learned about the safety and effectiveness of these promising new therapies by studying them first in non-human primates before initiating clinical trials, as shown by the results of a study published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers.

29 Dec 2016

Researchers use CRISPR-based genetic screening to identify three promising HIV treatment targets

Investigators from Whitehead Institute, the Ragon Institute of MGH, MIT and Harvard and the Broad Institute of MIT and Harvard have used CRISPR-Cas9 gene-editing technology to identify three promising new targets for treatment of HIV infection.

20 Dec 2016

Gene Therapy News and Research

Further Reading

New report highlights serious health problems faced by spinal cord injury patients

Gene therapy treats myotubular myopathy in dogs

Signals from fat cells may aid in diagnostics for metabolic conditions

Penn Medicine's ODC collaborates with FAST to develop gene therapy for Angelman syndrome

Scientists identify how imbalance of calcium in mitochondria may contribute to Alzheimer's disease

Review shows lipid nanoparticles as highly promising delivery systems in gene therapy

Northwestern Medicine scientists discover retinal cell that may cause myopia

Research unravels molecular mechanisms of antisense oligonucleotides

Study reveals new molecular mechanism vital for associative memory encoding in the hippocampus

Scientists restore partial hearing and balance in deaf mice using new gene therapy

New MIP system for therapeutic gene delivery can enhance transgene expression using AAV vector

New gene therapy for Pompe disease may replace currently available treatments

Novel gene therapy restores partial hearing and balance in mice born with genetic deafness

Salk Institute researchers reveal how to curb immune enthusiasm

MedCision announce participation at upcoming conference on cell and gene therapy

Orphan Disease Center establishes new initiative that focuses on ALS

New research suggests novel combination approach to fight against gliomas

AAV-based gene therapy to treat liver disorders advances into human testing

Studies in non-human primates provide more predictive results in genetic causes of blindness

Researchers use CRISPR-based genetic screening to identify three promising HIV treatment targets

Leveraging the power of automation to boost research

Automating research - solutions and best practices

How can microdialysis benefit drug development

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