Genetic Disorders

News Genetic Disorders

• A scanner for hereditary defects
  

  Our genetic material is constantly exposed to damage, which the body’s own proteins normally repair. One of these proteins works like a scanner, continually scouring the genetic material for signs of damage. Researchers from the Institute of Veterinary Pharmacology and Toxicology at the University o more

• New gene transfer strategy shows promise for limb girdle and other muscular dystrophies

  The challenge of treating patients with genetic disorders in which a single mutated gene is simply too large to be replaced using traditional gene therapy techniques may soon be a thing of the past. A Nationwide Children's Hospital study describes a new gene therapy approach capable of delivering fu more

• Scientists Make Groundbreaking Discovery Of Mutation Causing Genetic Disorder In Humans

  Scientists at A*STAR's Institute of Medical Biology (IMB), in collaboration with doctors and scientists in Jordan, Turkey, Switzerland and USA, have identified the genetic cause of a birth defect known as Hamamy syndrome. Their groundbreaking findings were published in Nature Genetics. The work lend more

Publications Genetic Disorders

• Mir‐142‐3p target sequences reduce transgene directed immunogenicity following intramuscular AAV1 vector‐mediated gene delivery

  Abstract Background Muscle represents an important tissue target for adeno‐associated virus (AAV) vector‐mediated gene transfer in muscular, metabolic or blood related genetic disorders. However, several studies demonstrated the appearance of immune responses against the transgene product afte more

• Autism Spectrum Disorders: The Quest for Genetic Syndromes

  Abstract Autism spectrum disorders (ASD) are a heterogeneous group of neurodevelopmental disabilities with various etiologies, but with a heritability estimate of more than 90%. Although the strong correlation between autism and genetic factors has been long established, the exact genetic backg more

• PhenoTips: Patient Phenotyping Software for Clinical and Research Use

  ABSTRACT We have developed PhenoTips: open source software for collecting and analyzing phenotypic information for patients with genetic disorders. Our software combines an easy‐to‐use interface, compatible with any device that runs a Web browser, with a standardized database back‐end. The Pheno more

Market studies Genetic Disorders

• Orphan Disease Therapeutics in Genetic Disorders to 2018 - Emerging Agents in Cystic Fibrosis Offer Strong Opportunities for Investment and Licensing Activity

  GBI Research, the leading business intelligence provider, has released its latest research, “Orphan Disease Therapeutics in Genetic Disorders to 2018 - Emerging Agents in Cystic Fibrosis Offer Strong Opportunities for Investment and Licensing Activity”, which provides insights into the global orphan more

• Protalix BioTherapeutics, Inc. (PLX) - Financial and Strategic SWOT Analysis Review

  Protalix BioTherapeutics, Inc. (Protalix) is a publicly held biopharmaceutical company. It is engaged in the development and supplying of recombinant therapeutic proteins based on its proprietary production system, ProCellEx (protein expression system). Protalix is focused on therapeutic proteins fo more

• Innovations in Protein Kinase Therapies - Significant Proportion of Protein Kinase Pipeline Contains Small Molecule Drugs That Target Oncology

  GBI Research’s report, “Innovations in Protein Kinase Therapies - Significant Proportion of Protein Kinase Pipeline Contains Small Molecule Drugs That Target Oncology”, provides in-depth analysis of innovations in protein kinase therapies in the current pharmaceutical market. The report analyzes the more

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