Sangamo Therapeutics and Pfizer team up for development of Hemophilia A gene therapy
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  May 18, 2017  
  Genetics  
  The latest Genetics news from News Medical  
 Sangamo Therapeutics and Pfizer team up for development of Hemophilia A gene therapySangamo Therapeutics and Pfizer team up for development of Hemophilia A gene therapy
 
Sangamo Therapeutics, Inc. and Pfizer Inc. today announced an exclusive, global collaboration and license agreement for the development and commercialization of gene therapy programs for Hemophilia A, including SB-525, one of Sangamo's four lead product candidates, which Sangamo expects will enter the clinic this quarter.
 
 
 Spanish scientists develop promising gene therapy method to treat Wilson's diseaseSpanish scientists develop promising gene therapy method to treat Wilson's disease
 
Scientists at the Center for Applied Medical Research at the University of Navarra have designed a promising gene therapy method to treat Wilson's disease, a rare pathology caused by the lack of or malfunction of a gene.
 
   Experimental gene therapy proven safe for preserving vision of people with AMDExperimental gene therapy proven safe for preserving vision of people with AMD
 
In a small and preliminary clinical trial, Johns Hopkins researchers and their collaborators have shown that an experimental gene therapy that uses viruses to introduce a therapeutic gene into the eye is safe and that it may be effective in preserving the vision of people with wet age-related macular degeneration.
 
   Study identifies first genetic locus for anorexia nervosaStudy identifies first genetic locus for anorexia nervosa
 
A landmark study led by UNC School of Medicine researchers has identified the first genetic locus for anorexia nervosa and has revealed that there may also be metabolic underpinnings to this potentially deadly illness.
 
   Scientists identify unusual mutations in endometriosis using gene sequencing toolsScientists identify unusual mutations in endometriosis using gene sequencing tools
 
Using gene sequencing tools, scientists from Johns Hopkins Medicine and the University of British Columbia have found a set of genetic mutations in samples from 24 women with benign endometriosis, a painful disorder marked by the growth of uterine tissue outside of the womb.
 
 New gene variant linked to Lou Gehrig's disease
 
New gene variant linked to Lou Gehrig's diseaseFor the first time, a variant in UBQLN4 gene has been associated with Lou Gehrig's disease or amyotrophic lateral sclerosis (ALS) - a progressive disease resulting in the loss of nerve cells that control muscle movement, which eventually leads to paralysis and death.
 
 
 Early-onset pancreatitis in children linked to family history and genetic mutations
 
Early-onset pancreatitis in children linked to family history and genetic mutationsA study published today in The Journal of Pediatrics suggests that early-onset pancreatitis in children is strongly associated with certain genetic mutations and family history of pancreatitis.
 
 
 Researchers discover genetic mutation that can cause aortic dissection
 
Researchers discover genetic mutation that can cause aortic dissectionResearchers at Umeå University in Sweden have discovered a genetic mutation that can cause dissection of the thoracic aorta, which is the body's main artery.
 
 
 Preclinical data reinforces potential efficacy, durability of investigational gene therapy for DMD
 
Preclinical data reinforces potential efficacy, durability of investigational gene therapy for DMDSolid Biosciences announced today that new data from two preclinical studies reinforce the potential of its investigational microdystrophin gene therapy, SGT-001, to be an effective treatment approach for Duchenne muscular dystrophy.
 
 
 Researchers identify many dangerous strains of TB pathogens
 
Researchers identify many dangerous strains of TB pathogensResearchers from the Vavilov Institute of General Genetics of the Russian Academy of Sciences (VIGG) and the Moscow Institute of Physics and Technology have established a catalog of mutations in 319 virulence genes of mycobacteria that cause tuberculosis.
 
 
 Micronic launches storage tube for low volume genomics
 
Micronic launches storage tube for low volume genomicsTo address the ever-increasing need for low volume genomics, Micronic launches the 0.30ml tube with external thread that enables miniaturization of reaction volume required in genomic applications. The tube cuts out intermediate (screening) plates and is more accessible for low volume liquid handlers. In addition, the automation friendly 0.
 
 
 Discovery of steroid hormone in fruit flies may shed new light on maternal hypothyroidism
 
Discovery of steroid hormone in fruit flies may shed new light on maternal hypothyroidismUniversity of Oregon researchers using Drosophila have identified a steroid hormone that triggers a vital transition in early brain development in which neural stem cells properly change gears to produce different kinds of neurons.
 
 
 Analyzing single-cell RNA data in pre-malignant tumors
 
Analyzing single-cell RNA data in pre-malignant tumorsRNA sequencing is basically the isolation of RNA from cells and the use of reverse transcriptase to turn the RNA into DNA. You can then use your standard DNA sequencing technologies to quantify the cDNA that you obtained from the reverse transcription reaction.
 
 
 Scientists develop world's first smartphone app to collect large data for people with MS
 
Scientists develop world's first smartphone app to collect large data for people with MSWhat if a single smartphone app could help solve the enigma of multiple sclerosis and move new treatments ahead at lightning speed? That was the bold idea that led Keck Medicine of USC neurologist Daniel Pelletier, MD, professor of neurology at the Keck School of Medicine of USC and division chief of the Neuro-Immunology and USC Multiple Sclerosis Center, to develop myMS, the world's first smartphone app capable of collecting large amounts of...