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    Gene Therapy

    Gene Therapy: Gene therapy is designed to introduce genetic material into cells to compensate for abnormal genes or to make a beneficial protein. If a mutated gene causes a necessary protein to be faulty or missing, gene therapy may be able to introduce a normal copy of the gene to restore the function of the protein.

    Trending News

    • JUL 26, 2019
      Genetics & Genomics
      Same Repetitive DNA Sequence at the Root of Several Rare Neurodegenerative Disorders
      Same Repetitive DNA Sequence at the Root of Several Rare Neurodegenerative Disorders
      Reporting in Nature Genetics, scientists have now identified a genetic feature underlying three neurodegenerative diseas ...
      Written By: Carmen Leitch
      2
      JUL 24, 2019
      Cell & Molecular Biology
      Could we be allergic to CRISPR?
      Could we be allergic to CRISPR?
      A new study published in Nature reveals an immunity to the CRISPR-Cas9 proteins in humans
      Written By: Carly Boyd
      2
      JUL 23, 2019
      Cell & Molecular Biology
      Exosomes are Involved in Brain Development
      Exosomes are Involved in Brain Development
      Researchers have learned more about the role of exosomes in neurodevelopment.
      Written By: Carmen Leitch
      1
      JUL 22, 2019
      Cancer
      Understanding osteosarcoma in kids and dogs
      Understanding osteosarcoma in kids and dogs
      New research published in the Nature journal, Communications Biology, shows evidence for a comparative study that scient ...
      Written By: Kathryn DeMuth Sullivan
      2
      JUL 19, 2019
      Cancer
      New hope for metastatic cancer treatment
      New hope for metastatic cancer treatment
      New research recently published in the journal iScience takes a deeper look into metastatic cancers, finding that a part ...
      Written By: Kathryn DeMuth Sullivan
      2
      JUL 16, 2019
      Genetics & Genomics
      Lipid Nanoparticles Improve the Delivery of CRISPR/Cas9 Into Cells
      Lipid Nanoparticles Improve the Delivery of CRISPR/Cas9 Into Cells
      The CRISPR/Cas9 gene editing tool has revolutionized biomedical research and has tremendous potential for use in the cli ...
      Written By: Carmen Leitch
      1
      JUL 09, 2019
      Cardiology
      Cardiovascular Risks And Ehlers-Danlos Syndrome
      Cardiovascular Risks And Ehlers-Danlos Syndrome
      Ehlers-Danlos Syndrome (EDS) affects the body’s connective tissues. These tissues are proteins that provide suppor ...
      Written By: Abbie Arce
      1
      JUL 08, 2019
      Genetics & Genomics
      A CRISPR System can Edit Only a Defective Copy of a Gene
      A CRISPR System can Edit Only a Defective Copy of a Gene
      With a more precise version of CRISPR/Cas9, researchers altered a single, mutated copy of a gene, leaving the correct on ...
      Written By: Carmen Leitch
      1
      JUL 04, 2019
      Genetics & Genomics
      Combination Therapy Eliminates HIV From Rodent Model
      Combination Therapy Eliminates HIV From Rodent Model
      In 2017, 1.8 million new cases of HIV infection were diagnosed around the world, and around 37 million people live with ...
      Written By: Carmen Leitch
      1
      JUL 03, 2019
      Genetics & Genomics
      Treating Psychosis by Targeting a Genetic Mutation
      Treating Psychosis by Targeting a Genetic Mutation
      It may one day be possible to treat psychiatric disorders with therapy that targets genetic mutations, which was done in ...
      Written By: Carmen Leitch
      2
      JUN 26, 2019
      Genetics & Genomics
      How a Genetically-modified Virus can Combat Prostate Cancer
      How a Genetically-modified Virus can Combat Prostate Cancer
      Around one in nine men will get prostate cancer, which is the second-deadliest cancer for American men after lung cancer ...
      Written By: Carmen Leitch
      1
      JUN 24, 2019
      Genetics & Genomics
      Genetic Predictors of a Failing Heart
      Genetic Predictors of a Failing Heart
      Scientists have discovered a gene, PPP1R3A, that may be the ultimate protection against heart failure.  Evidence sh ...
      Written By: Amanda Mikyska
      1
      JUN 19, 2019
      Genetics & Genomics
      Research Reveals Role of Leptin in Human Appetite
      Research Reveals Role of Leptin in Human Appetite
        A drop in leptin concentration, a hormone released from fat stores, was vaguely understood to influence increased ...
      Written By: Amanda Mikyska
      1
      JUN 19, 2019
      Genetics & Genomics
      Genetic Link to Heart Disease is Stronger Than Thought
      Genetic Link to Heart Disease is Stronger Than Thought
      Previous work did not look at the impact of small changes in regulatory genes.
      Written By: Carmen Leitch
      1
      JUN 11, 2019
      Technology
      Artificial Intelligence Can Help Detect Rare Diseases
      Artificial Intelligence Can Help Detect Rare Diseases
      An estimated half a million children worldwide are born with a rare hereditary disease. To obtain a definitive diagnosis ...
      Written By: Nouran Amin
      1
      JUN 10, 2019
      Genetics & Genomics
      CRISPR 2.0: Using Transposons to Improve and Expand CRISPR Application
      CRISPR 2.0: Using Transposons to Improve and Expand CRISPR Application
      Source: Science CRISPR-cas9, commonly referred to simply as CRISPR, is a bacterial immune system that was first used as ...
      Written By: Amanda Mikyska
      4
      JUN 07, 2019
      Health & Medicine
      In Vivo Tissue Stem Cell and Progenitor Cell Genome Modification
      In Vivo Tissue Stem Cell and Progenitor Cell Genome Modification
      A stem cell, by definition, must be able to self-replicate to make more stem cells and to differentiate into a matu ...
      Written By: Dena Aruta
      1
      JUN 03, 2019
      Genetics & Genomics
      CRISPR Babies May Face Shortened Lifespan
      CRISPR Babies May Face Shortened Lifespan
      Genetic mutations can lead to disease, and scientists have been searching for ways to effectively and safely repair such ...
      Written By: Carmen Leitch
      1
      JUN 01, 2019
      Genetics & Genomics
      How Cas13 Works in Bacteria
      How Cas13 Works in Bacteria
      Cas13 makes a great diagnostic tool, and now researchers know why it naturally makes so many cuts in RNA.
      Written By: Carmen Leitch
      1
      MAY 31, 2019
      Microbiology
      Another Study Links the Gut Microbiome to Autism
      Another Study Links the Gut Microbiome to Autism
      As of 2018, around one in five 8-year-olds were impacted by autism spectrum disorder.
      Written By: Carmen Leitch
      1
      MAY 29, 2019
      Health & Medicine
      Novel Gene Therapy Approved by FDA to Treat Spinal Muscular Atrophy in Pediatric Patients
      Novel Gene Therapy Approved by FDA to Treat Spinal Muscular Atrophy in Pediatric Patients
      The most common form of spinal muscular atrophy (SMA), a rare genetic disease, is caused by mutations of chromosome ...
      Written By: Dena Aruta
      1
      MAY 27, 2019
      Genetics & Genomics
      Watching the Transfer of Drug Resistance in Bacteria
      Watching the Transfer of Drug Resistance in Bacteria
      Using fluorescence, researchers visualized the spread of antibiotic resistance among bacteria - in the presence of antib ...
      Written By: Carmen Leitch
      2
      MAY 26, 2019
      Cell & Molecular Biology
      A Test to Measure the Effectiveness of Stem Cell Transplantation
      A Test to Measure the Effectiveness of Stem Cell Transplantation
      Stem cells-based therapies may one day repair disorders caused by damaged cells. But those therapeutics must be tested.
      Written By: Carmen Leitch
      2
      MAY 24, 2019
      Microbiology
      Studies Confirm That a Poor Diet can Lead to Disease
      Studies Confirm That a Poor Diet can Lead to Disease
      Recent work has shown that people all over the world can improve their health and lower cancer risk by eating a more bal ...
      Written By: Carmen Leitch
      1
    JUL 26, 2019
    Genetics & Genomics
    Same Repetitive DNA Sequence at the Root of Several Rare Neurodegenerative Disorders
    Same Repetitive DNA Sequence at the Root of Several Rare Neurodegenerative Disorders
    Reporting in Nature Genetics, scientists have now identified a genetic feature underlying three neurodegenerative diseas ...
    Written By: Carmen Leitch
    2
    JUL 24, 2019
    Cell & Molecular Biology
    Could we be allergic to CRISPR?
    Could we be allergic to CRISPR?
    A new study published in Nature reveals an immunity to the CRISPR-Cas9 proteins in humans
    Written By: Carly Boyd
    2
    JUL 23, 2019
    Cell & Molecular Biology
    Exosomes are Involved in Brain Development
    Exosomes are Involved in Brain Development
    Researchers have learned more about the role of exosomes in neurodevelopment.
    Written By: Carmen Leitch
    1
    JUL 22, 2019
    Cancer
    Understanding osteosarcoma in kids and dogs
    Understanding osteosarcoma in kids and dogs
    New research published in the Nature journal, Communications Biology, shows evidence for a comparative study that scient ...
    Written By: Kathryn DeMuth Sullivan
    2
    JUL 19, 2019
    Cancer
    New hope for metastatic cancer treatment
    New hope for metastatic cancer treatment
    New research recently published in the journal iScience takes a deeper look into metastatic cancers, finding that a part ...
    Written By: Kathryn DeMuth Sullivan
    2
     
     
  • JUL 16, 2019
    Genetics & Genomics
    Lipid Nanoparticles Improve the Delivery of CRISPR/Cas9 Into Cells
    Lipid Nanoparticles Improve the Delivery of CRISPR/Cas9 Into Cells
    The CRISPR/Cas9 gene editing tool has revolutionized biomedical research and has tremendous potential for use in the cli ...
    Written By: Carmen Leitch
    1
    JUL 09, 2019
    Cardiology
    Cardiovascular Risks And Ehlers-Danlos Syndrome
    Cardiovascular Risks And Ehlers-Danlos Syndrome
    Ehlers-Danlos Syndrome (EDS) affects the body’s connective tissues. These tissues are proteins that provide suppor ...
    Written By: Abbie Arce
    1
    JUL 08, 2019
    Genetics & Genomics
    A CRISPR System can Edit Only a Defective Copy of a Gene
    A CRISPR System can Edit Only a Defective Copy of a Gene
    With a more precise version of CRISPR/Cas9, researchers altered a single, mutated copy of a gene, leaving the correct on ...
    Written By: Carmen Leitch
    1
    JUL 04, 2019
    Genetics & Genomics
    Combination Therapy Eliminates HIV From Rodent Model
    Combination Therapy Eliminates HIV From Rodent Model
    In 2017, 1.8 million new cases of HIV infection were diagnosed around the world, and around 37 million people live with ...
    Written By: Carmen Leitch
    1
    JUL 03, 2019
    Genetics & Genomics
    Treating Psychosis by Targeting a Genetic Mutation
    Treating Psychosis by Targeting a Genetic Mutation
    It may one day be possible to treat psychiatric disorders with therapy that targets genetic mutations, which was done in ...
    Written By: Carmen Leitch
    2
     
     
  • JUN 26, 2019
    Genetics & Genomics
    How a Genetically-modified Virus can Combat Prostate Cancer
    How a Genetically-modified Virus can Combat Prostate Cancer
    Around one in nine men will get prostate cancer, which is the second-deadliest cancer for American men after lung cancer ...
    Written By: Carmen Leitch
    1
    JUN 24, 2019
    Genetics & Genomics
    Genetic Predictors of a Failing Heart
    Genetic Predictors of a Failing Heart
    Scientists have discovered a gene, PPP1R3A, that may be the ultimate protection against heart failure.  Evidence sh ...
    Written By: Amanda Mikyska
    1
    JUN 19, 2019
    Genetics & Genomics
    Research Reveals Role of Leptin in Human Appetite
    Research Reveals Role of Leptin in Human Appetite
      A drop in leptin concentration, a hormone released from fat stores, was vaguely understood to influence increased ...
    Written By: Amanda Mikyska
    1
    JUN 19, 2019
    Genetics & Genomics
    Genetic Link to Heart Disease is Stronger Than Thought
    Genetic Link to Heart Disease is Stronger Than Thought
    Previous work did not look at the impact of small changes in regulatory genes.
    Written By: Carmen Leitch
    1
    JUN 11, 2019
    Technology
    Artificial Intelligence Can Help Detect Rare Diseases
    Artificial Intelligence Can Help Detect Rare Diseases
    An estimated half a million children worldwide are born with a rare hereditary disease. To obtain a definitive diagnosis ...
    Written By: Nouran Amin
    1
     
     
  • JUN 10, 2019
    Genetics & Genomics
    CRISPR 2.0: Using Transposons to Improve and Expand CRISPR Application
    CRISPR 2.0: Using Transposons to Improve and Expand CRISPR Application
    Source: Science CRISPR-cas9, commonly referred to simply as CRISPR, is a bacterial immune system that was first used as ...
    Written By: Amanda Mikyska
    4
    JUN 07, 2019
    Health & Medicine
    In Vivo Tissue Stem Cell and Progenitor Cell Genome Modification
    In Vivo Tissue Stem Cell and Progenitor Cell Genome Modification
    A stem cell, by definition, must be able to self-replicate to make more stem cells and to differentiate into a matu ...
    Written By: Dena Aruta
    1
    JUN 03, 2019
    Genetics & Genomics
    CRISPR Babies May Face Shortened Lifespan
    CRISPR Babies May Face Shortened Lifespan
    Genetic mutations can lead to disease, and scientists have been searching for ways to effectively and safely repair such ...
    Written By: Carmen Leitch
    1
    JUN 01, 2019
    Genetics & Genomics
    How Cas13 Works in Bacteria
    How Cas13 Works in Bacteria
    Cas13 makes a great diagnostic tool, and now researchers know why it naturally makes so many cuts in RNA.
    Written By: Carmen Leitch
    1
    MAY 31, 2019
    Microbiology
    Another Study Links the Gut Microbiome to Autism
    Another Study Links the Gut Microbiome to Autism
    As of 2018, around one in five 8-year-olds were impacted by autism spectrum disorder.
    Written By: Carmen Leitch
    1
     
     
  • MAY 29, 2019
    Health & Medicine
    Novel Gene Therapy Approved by FDA to Treat Spinal Muscular Atrophy in Pediatric Patients
    Novel Gene Therapy Approved by FDA to Treat Spinal Muscular Atrophy in Pediatric Patients
    The most common form of spinal muscular atrophy (SMA), a rare genetic disease, is caused by mutations of chromosome ...
    Written By: Dena Aruta
    1
    MAY 27, 2019
    Genetics & Genomics
    Watching the Transfer of Drug Resistance in Bacteria
    Watching the Transfer of Drug Resistance in Bacteria
    Using fluorescence, researchers visualized the spread of antibiotic resistance among bacteria - in the presence of antib ...
    Written By: Carmen Leitch
    2
    MAY 26, 2019
    Cell & Molecular Biology
    A Test to Measure the Effectiveness of Stem Cell Transplantation
    A Test to Measure the Effectiveness of Stem Cell Transplantation
    Stem cells-based therapies may one day repair disorders caused by damaged cells. But those therapeutics must be tested.
    Written By: Carmen Leitch
    2
    MAY 24, 2019
    Microbiology
    Studies Confirm That a Poor Diet can Lead to Disease
    Studies Confirm That a Poor Diet can Lead to Disease
    Recent work has shown that people all over the world can improve their health and lower cancer risk by eating a more bal ...
    Written By: Carmen Leitch
    1
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