New Gene Therapy Gives Hope To People With Inherited Eye Disorder

The first successful gene therapy for an inherited form of blindness was recently reviewed by an expert panel of advisers at US Food and Drug Administration (FDA). They unanimously voted in recommendation of the treatment. The FDA now has until January 12, 2018 to approve the treatment. Spark Therapeutics, an…Continue readingNew Gene Therapy Gives Hope To People With Inherited Eye Disorder

Phospholipid Scramblase 1 Regulates Microglia Viral Immune Response

Inhibiting a protein called phospholipid scramblase 1 (PLSCR1) controls infected cell’s antiviral responses and provides long-term protection from immune attack and excessive inflammation, researchers at Salk Institute have discovered. The finding holds promise both for virally delivered treatments and inflammatory conditions like infections; autoimmune disorders such as lupus; or neurodegenerative…Continue readingPhospholipid Scramblase 1 Regulates Microglia Viral Immune Response

Sickle-Cell Gene Therapy From Bluebird Bio Appears Encouraging In Early Results

The first patient with severe sickle cell disease (SCD) to be treated with LentiGlobin BB305, produced by Bluebird Bio, seems to working effectively so far, researchers said today. LentiGlobin BB305 works by correcting the underlying gene defect which causes sickle-cell disease. It helps the body make enough functional hemoglobin to…Continue readingSickle-Cell Gene Therapy From Bluebird Bio Appears Encouraging In Early Results

New Pluripotent Stem Cell Form Is Region Specific

A new type of pluripotent stem cell has been discovered by scientists at the Salk Institute. The cells’ identity is linked to their location in a developing embryo. The stem cells usually used in scientific study have an identity linked to their time-related stage of development. Pluripotent stem cell are…Continue readingNew Pluripotent Stem Cell Form Is Region Specific

Harmless Artificial Virus Developed For Gene Therapy

A team of scientists from Universitat Autònoma de Barcelona has produced an alternative to the use of viruses in gene therapy. The researchers synthesised nanoparticles which act as artificial viruses, capable of surrounding DNA fragments and releasing them as therapeutic agents, with no biological risk, into the interior of the…Continue readingHarmless Artificial Virus Developed For Gene Therapy

New Gene Therapy For Hemophilia Shows Potential As Safe Treatment

A multi-year, ongoing study suggests that a new kind of gene therapy for hemophilia B could be safe and effective for human patients. The research showed that a reprogrammed retrovirus could successfully transfer new factor IX (clotting) genes into animals with hemophilia B to dramatically decrease spontaneous bleeding. Thus far,…Continue readingNew Gene Therapy For Hemophilia Shows Potential As Safe Treatment