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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.
NYU dentists receive grant to test whether non-viral gene delivery can effectively treat oral cancer pain

NYU dentists receive grant to test whether non-viral gene delivery can effectively treat oral cancer pain

The National Institute of Dental and Craniofacial Research, part of the National Institutes of Health, has awarded Drs. Brian Schmidt and Seiichi Yamano a $1.2M (3-year) grant to test whether their non-viral gene delivery method can effectively and safely treat oral cancer pain. [More]
New book highlights advances, challenges in development of nanotheranostics for personalized medicine

New book highlights advances, challenges in development of nanotheranostics for personalized medicine

The future of medicine is now dawning upon us.Nanomedicines demonstrate the capability to enhance drug properties by offering protection from degradation, enabling controlled release and biodistribution and increasing bioavailability. [More]
Gamma-ray irradiation improves safety of stem cell transplantation in Parkinson’s disease patients

Gamma-ray irradiation improves safety of stem cell transplantation in Parkinson’s disease patients

Replacing dopamine-producing cells in the brain represents a promising therapeutic approach in Parkinson's disease, and a new study shows how post-transplantation gamma-ray irradiation can reduce the risk of tumor formation. [More]
Lack of Alix protein leads to occurrence of hydrocephalus in the brain

Lack of Alix protein leads to occurrence of hydrocephalus in the brain

A team led by researchers at St. Jude Children's Research Hospital report that mice lacking the protein Alix develop hydrocephalus or "water on the brain." Alix ensures that epithelial cells of the choroid plexus are oriented correctly with respect to one another to prevent compromise of the epithelial barrier. [More]
Scientists develop non-toxic transplantation procedure using antibodies to target blood stem cells in mice

Scientists develop non-toxic transplantation procedure using antibodies to target blood stem cells in mice

Harvard Stem Cell Institute scientists have taken the first steps toward developing a treatment that would make bone marrow - blood stem cell - transplantation safer and, as a result, more widely available to the millions of people living with blood disorders like sickle cell anemia, thalassemia, and AIDS. [More]
Scientists use new technique to repair fibrotic liver cells within the organ

Scientists use new technique to repair fibrotic liver cells within the organ

Advances in stem cell research have made it possible to convert patients' skin cells into heart cells, kidney cells, liver cells and more in the lab dish, giving researchers hope that one day such cells could replace organ transplantation for patients with organ failure. [More]
New FDA draft guidelines place tighter restrictions on surgical practices using tissue-based regenerative therapies

New FDA draft guidelines place tighter restrictions on surgical practices using tissue-based regenerative therapies

The therapeutic use of human cell and tissue products is highly regulated by the U.S. government, but a specific exception allows surgeons to harvest, manipulate, and implant tissues in many commonly performed procedures. [More]
Increasing specific microRNA levels can restore chemotherapy sensitivity in pancreatic cancer cells

Increasing specific microRNA levels can restore chemotherapy sensitivity in pancreatic cancer cells

By increasing the level of a specific microRNA (miRNA) molecule, researchers have for the first time restored chemotherapy sensitivity in vitro to a line of human pancreatic cancer cells that had developed resistance to a common treatment drug. [More]
Scientists debate role of AAV in causing hepatocellular carcinoma

Scientists debate role of AAV in causing hepatocellular carcinoma

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. [More]
CGT, CTM CRC collaborate to test patented scaffold technology for commercial scale cell expansion systems

CGT, CTM CRC collaborate to test patented scaffold technology for commercial scale cell expansion systems

The Cell and Gene Therapy Catapult and the CRC for Cell Therapy Manufacturing, the Australian centre for translation of cell therapy technologies, today announce a project to test at scale CTM CRC’s patented scaffold technology. [More]
Gene editing technology helps excise segment of HIV-1 DNA from genomes of living animals

Gene editing technology helps excise segment of HIV-1 DNA from genomes of living animals

Using gene editing technology, researchers at the Lewis Katz School of Medicine at Temple University have, for the first time, successfully excised a segment of HIV-1 DNA - the virus responsible for AIDS - from the genomes of living animals. [More]
Nanoparticle-based gene therapy may hold promise for patients with asthma

Nanoparticle-based gene therapy may hold promise for patients with asthma

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. [More]
CGT, University of Aberdeen announce creation of new company to develop novel technology for diabetes treatment

CGT, University of Aberdeen announce creation of new company to develop novel technology for diabetes treatment

The Cell and Gene Therapy Catapult and University of Aberdeen, UK, today announce the creation of Islexa, a new company developing a novel technology to produce laboratory grown islets, the organoids responsible for insulin production. [More]
Researchers develop novel ss-siRNAs to turn off disease-related genes

Researchers develop novel ss-siRNAs to turn off disease-related genes

Combining the therapeutic potential and advantages of existing oligonucleotide-based approaches to turn off disease-related genes, a type of single-stranded silencing RNAs (ss-siRNAs) has shown significantly improved potency and activity. [More]
Gene therapy shows promise in treating glioblastoma

Gene therapy shows promise in treating glioblastoma

A team from the International School for Advanced Studies in Trieste has obtained very promising results by applying gene therapy to glioblastoma. [More]
Researchers agree to test new stem cell gene therapy for Sanfilippo disease in human trial

Researchers agree to test new stem cell gene therapy for Sanfilippo disease in human trial

Scientists in Manchester, who have developed a stem cell gene therapy to reverse a fatal childhood illness, have agreed to work with a new therapeutics company to test it in a human trial. [More]
Patient-specific immunotherapeutic vaccines may improve overall survival of melanoma patients

Patient-specific immunotherapeutic vaccines may improve overall survival of melanoma patients

Two patients with melanoma that had spread to the liver survived for at least 8.5 and 12 years after resection of the hepatic tumor and treatment with patient-specific immunotherapeutic vaccines. [More]
Experimental therapy stops glioblastoma, high-grade gliomas in human cells and mouse models

Experimental therapy stops glioblastoma, high-grade gliomas in human cells and mouse models

Researchers report in the journal Cancer Cell an experimental therapy that in laboratory tests on human cells and mouse models stops aggressive, treatment-resistant and deadly brain cancers called glioblastoma and high-grade gliomas. [More]
Researchers identify new molecular targets for recovering motor function in men afflicted with SBMA

Researchers identify new molecular targets for recovering motor function in men afflicted with SBMA

Michigan State University researchers used an old-fashioned neurobiology technique to explore new avenues for treatments to reverse a late-onset neurodegenerative disease that robs men of the capacity to walk, run, chew and swallow. [More]
Unique attributes of zebrafish may help study human blood disorders

Unique attributes of zebrafish may help study human blood disorders

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 normal hematopoetic development in humans as well as blood disorders and malignancies, as described in a Review article in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. [More]
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