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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

    • AUG 20, 2025
      Genetics & Genomics
      A Non-Viral Gene Therapy for Heart Disease
      A Non-Viral Gene Therapy for Heart Disease
      Heart diseases are still the number one cause of death worldwide. But scientists are trying to change that...
      Written By: Carmen Leitch
      4
      AUG 04, 2025
      Cell & Molecular Biology
      A Novel Gene Therapy for Osteoarthritis Pain
      A Novel Gene Therapy for Osteoarthritis Pain
      Genetic errors can cause many different diseases, and scientists have been looking for methods to fix those errors for d ...
      Written By: Carmen Leitch
      5
      APR 14, 2025
      eBooks
      Optimizing Gene Therapy: A Faster AAV Analysis Method
      Input your information to
      to access this free eBook
      Optimizing Gene Therapy: A Faster AAV Analysis Method
      Adeno-associated virus (AAV) gene therapies hold great promise, but ensuring product quality is critical.
      Sponsored By: Sartorius
      7
      APR 01, 2025
      Drug Discovery & Development
      Top Transfection Tips for Enhanced Success
      Top Transfection Tips for Enhanced Success
       
      Sponsored By: Sartorius
      4
      FEB 19, 2025
      Genetics & Genomics
      Assessing Gene Therapy Approaches for Down Syndrome
      Assessing Gene Therapy Approaches for Down Syndrome
      Decades ago, researchers showed that Down syndrome (also known as trisomy 21), was caused when individuals carried an ex ...
      Written By: Carmen Leitch
      4
      DEC 03, 2024
      Immunology
      Asia-Pacific Precision Oncology Summit 2024
      Asia-Pacific Precision Oncology Summit 2024
      Join us to explore the latest insights into the future of precision oncology! This year, the Agilent Precision Oncology ...
      Sponsored By: Agilent
      4
      NOV 17, 2024
      Genetics & Genomics
      A New Approach in Usher Syndrome Gene Therapy
      A New Approach in Usher Syndrome Gene Therapy
      Delivering very large genes to cells can pose a serious challenge, so scientists developed a new method.
      Written By: Carmen Leitch
      3
      OCT 29, 2024
      Genetics & Genomics
      A New Gene Therapy Technique Aims to Deliver Treatment Before Birth
      A New Gene Therapy Technique Aims to Deliver Treatment Before Birth
      Researchers have developed a novel delivery system for sending CRISPR reagents to brain cells, where it performs its gen ...
      Written By: Carmen Leitch
      4
      SEP 14, 2024
      Technology
      Gene Therapy Significantly Improves Eyesight
      Gene Therapy Significantly Improves Eyesight
      A team of researchers report findings from a Phase I/II gene therapy clinical trial for patients with Leber congenital a ...
      Written By: Ryan Vingum
      3
      AUG 20, 2024
      Neuroscience
      New Treatment for Dry AMD Shows Promise in Vision Improvement
      New Treatment for Dry AMD Shows Promise in Vision Improvement
      Age-related macular degeneration (AMD) is a leading cause of vision loss among people aged 50 and older. While treatment ...
      Written By: Greta Anne
      4
      JUL 23, 2024
      Genetics & Genomics
      A New Approach in Gene Therapy for Muscular Dystrophy
      A New Approach in Gene Therapy for Muscular Dystrophy
      Scientists have created a new gene therapy for Duchenne muscular dystrophy that may not only help stop the disease in MD ...
      Written By: Carmen Leitch
      5
      MAY 14, 2024
      Cell & Molecular Biology
      A Gene Editing Treatment That Takes Aim at Herpes Infections
      A Gene Editing Treatment That Takes Aim at Herpes Infections
      It's estimated that about 3.7 billion people under the age of 50 are infected with (HSV-1), which can cause oral herpes.
      Written By: Carmen Leitch
      4
      MAY 01, 2024
      Genetics & Genomics
      The Complex Genetics of a Rare Disorder are Deciphered
      The Complex Genetics of a Rare Disorder are Deciphered
      Researchers have been trying to solve the mystery of a rare genetic disorder called spinocerebellar ataxia 4 (SCA4) for ...
      Written By: Carmen Leitch
      4
      APR 26, 2024
      Genetics & Genomics
      Deciphering the Complexities of an Autism & Seizure-Linked Gene
      Deciphering the Complexities of an Autism & Seizure-Linked Gene
      Mutations in a gene called SCN2A are known to cause seizures in infants, and have been associated with autism spectrum d ...
      Written By: Carmen Leitch
      5
      APR 18, 2024
      Immunology
      Successful Immunotherapy Outcome for Rare Eye Cancer
      Successful Immunotherapy Outcome for Rare Eye Cancer
      Highlights: A novel clinical tool discovered to predict patients’ response to therapy. Scientists took patient cel ...
      Written By: Brian G. Morreale
      3
      APR 02, 2024
      Neuroscience
      Giant Axonal Neuropathy: Promising Advances in Gene Therapy
      Giant Axonal Neuropathy: Promising Advances in Gene Therapy
      Giant axonal neuropathy (GAN) is a rare, autosomal recessive neurodegenerative disorder, characterized by a spectrum of ...
      Written By: Greta Anne
      3
      MAR 08, 2024
      Cell & Molecular Biology
      Scientists Identify a Kind of Genetic Anxiety Switch
      Scientists Identify a Kind of Genetic Anxiety Switch
      While it's normal to be a little anxious about stressful life events or problems, anxiety can also start to interfer ...
      Written By: Carmen Leitch
      7
      MAR 04, 2024
      Cell & Molecular Biology
      Cell and gene therapy production - improvements with single-use technologies
      Cell and gene therapy production - improvements with single-use technologies
      With astonishing advances in molecular biology, cell and gene therapies represent a new class of pharmaceuticals availab ...
      Sponsored By: Single Use Support
      4
      MAR 01, 2024
      Clinical & Molecular DX
      An Eye Test Can Detect Some Types of Autism
      An Eye Test Can Detect Some Types of Autism
      Autism is thought to affect about one of every 36 American children. New research has shown that it may be possible to d ...
      Written By: Carmen Leitch
      3
      FEB 29, 2024
      Immunology
      Protein Signal Drives Pro-Tumor Myeloid Genesis
      Protein Signal Drives Pro-Tumor Myeloid Genesis
      Myeloid cells are a population of cells classified to denote a specific lineage. “Myeloid” specifically refe ...
      Written By: Brian G. Morreale
      5
      FEB 29, 2024
      Immunology
      Novel Gene Editing Tool Improves Immune Cell Activity
      Novel Gene Editing Tool Improves Immune Cell Activity
      Jennifer Doudna and Emmanuelle Charpentier were awarded the 2020 Nobel Prize in Chemistry for their pioneering work in g ...
      Written By: Brian G. Morreale
      5
      FEB 15, 2024
      Immunology
      New Mechanism Reveals How Tumors Evade Immune Detection
      New Mechanism Reveals How Tumors Evade Immune Detection
      Scientists have dedicated their lives to finding a cure for cancer. America became a science hub for cancer research, pa ...
      Written By: Brian G. Morreale
      4
      FEB 06, 2024
      Genetics & Genomics
      Another CRISPR-Based Therapy Relieves Patients of Disease Symptoms
      Another CRISPR-Based Therapy Relieves Patients of Disease Symptoms
      The CRISPR gene editing tool was developed about a decade ago, and since then it has transformed research in the life sc ...
      Written By: Carmen Leitch
      4
      FEB 01, 2024
      Cancer
      Understanding the Links between Circadian Clock Tumor Growth
      Understanding the Links between Circadian Clock Tumor Growth
      The circadian clock describes the 24-hour internal clock in our brains.  The rhythms that regulate our awake and sl ...
      Written By: Katie Kokolus
      4
    AUG 20, 2025
    Genetics & Genomics
    A Non-Viral Gene Therapy for Heart Disease
    A Non-Viral Gene Therapy for Heart Disease
    Heart diseases are still the number one cause of death worldwide. But scientists are trying to change that...
    Written By: Carmen Leitch
    4
    AUG 04, 2025
    Cell & Molecular Biology
    A Novel Gene Therapy for Osteoarthritis Pain
    A Novel Gene Therapy for Osteoarthritis Pain
    Genetic errors can cause many different diseases, and scientists have been looking for methods to fix those errors for d ...
    Written By: Carmen Leitch
    5
    APR 14, 2025
    eBooks
    Optimizing Gene Therapy: A Faster AAV Analysis Method
    Input your information to
    to access this free eBook
    Optimizing Gene Therapy: A Faster AAV Analysis Method
    Adeno-associated virus (AAV) gene therapies hold great promise, but ensuring product quality is critical.
    Sponsored By: Sartorius
    7
    APR 01, 2025
    Drug Discovery & Development
    Top Transfection Tips for Enhanced Success
    Top Transfection Tips for Enhanced Success
     
    Sponsored By: Sartorius
    4
    FEB 19, 2025
    Genetics & Genomics
    Assessing Gene Therapy Approaches for Down Syndrome
    Assessing Gene Therapy Approaches for Down Syndrome
    Decades ago, researchers showed that Down syndrome (also known as trisomy 21), was caused when individuals carried an ex ...
    Written By: Carmen Leitch
    4
     
     
  • DEC 03, 2024
    Immunology
    Asia-Pacific Precision Oncology Summit 2024
    Asia-Pacific Precision Oncology Summit 2024
    Join us to explore the latest insights into the future of precision oncology! This year, the Agilent Precision Oncology ...
    Sponsored By: Agilent
    4
    NOV 17, 2024
    Genetics & Genomics
    A New Approach in Usher Syndrome Gene Therapy
    A New Approach in Usher Syndrome Gene Therapy
    Delivering very large genes to cells can pose a serious challenge, so scientists developed a new method.
    Written By: Carmen Leitch
    3
    OCT 29, 2024
    Genetics & Genomics
    A New Gene Therapy Technique Aims to Deliver Treatment Before Birth
    A New Gene Therapy Technique Aims to Deliver Treatment Before Birth
    Researchers have developed a novel delivery system for sending CRISPR reagents to brain cells, where it performs its gen ...
    Written By: Carmen Leitch
    4
    SEP 14, 2024
    Technology
    Gene Therapy Significantly Improves Eyesight
    Gene Therapy Significantly Improves Eyesight
    A team of researchers report findings from a Phase I/II gene therapy clinical trial for patients with Leber congenital a ...
    Written By: Ryan Vingum
    3
    AUG 20, 2024
    Neuroscience
    New Treatment for Dry AMD Shows Promise in Vision Improvement
    New Treatment for Dry AMD Shows Promise in Vision Improvement
    Age-related macular degeneration (AMD) is a leading cause of vision loss among people aged 50 and older. While treatment ...
    Written By: Greta Anne
    4
     
     
  • JUL 23, 2024
    Genetics & Genomics
    A New Approach in Gene Therapy for Muscular Dystrophy
    A New Approach in Gene Therapy for Muscular Dystrophy
    Scientists have created a new gene therapy for Duchenne muscular dystrophy that may not only help stop the disease in MD ...
    Written By: Carmen Leitch
    5
    MAY 14, 2024
    Cell & Molecular Biology
    A Gene Editing Treatment That Takes Aim at Herpes Infections
    A Gene Editing Treatment That Takes Aim at Herpes Infections
    It's estimated that about 3.7 billion people under the age of 50 are infected with (HSV-1), which can cause oral herpes.
    Written By: Carmen Leitch
    4
    MAY 01, 2024
    Genetics & Genomics
    The Complex Genetics of a Rare Disorder are Deciphered
    The Complex Genetics of a Rare Disorder are Deciphered
    Researchers have been trying to solve the mystery of a rare genetic disorder called spinocerebellar ataxia 4 (SCA4) for ...
    Written By: Carmen Leitch
    4
    APR 26, 2024
    Genetics & Genomics
    Deciphering the Complexities of an Autism & Seizure-Linked Gene
    Deciphering the Complexities of an Autism & Seizure-Linked Gene
    Mutations in a gene called SCN2A are known to cause seizures in infants, and have been associated with autism spectrum d ...
    Written By: Carmen Leitch
    5
    APR 18, 2024
    Immunology
    Successful Immunotherapy Outcome for Rare Eye Cancer
    Successful Immunotherapy Outcome for Rare Eye Cancer
    Highlights: A novel clinical tool discovered to predict patients’ response to therapy. Scientists took patient cel ...
    Written By: Brian G. Morreale
    3
     
     
  • APR 02, 2024
    Neuroscience
    Giant Axonal Neuropathy: Promising Advances in Gene Therapy
    Giant Axonal Neuropathy: Promising Advances in Gene Therapy
    Giant axonal neuropathy (GAN) is a rare, autosomal recessive neurodegenerative disorder, characterized by a spectrum of ...
    Written By: Greta Anne
    3
    MAR 08, 2024
    Cell & Molecular Biology
    Scientists Identify a Kind of Genetic Anxiety Switch
    Scientists Identify a Kind of Genetic Anxiety Switch
    While it's normal to be a little anxious about stressful life events or problems, anxiety can also start to interfer ...
    Written By: Carmen Leitch
    7
    MAR 04, 2024
    Cell & Molecular Biology
    Cell and gene therapy production - improvements with single-use technologies
    Cell and gene therapy production - improvements with single-use technologies
    With astonishing advances in molecular biology, cell and gene therapies represent a new class of pharmaceuticals availab ...
    Sponsored By: Single Use Support
    4
    MAR 01, 2024
    Clinical & Molecular DX
    An Eye Test Can Detect Some Types of Autism
    An Eye Test Can Detect Some Types of Autism
    Autism is thought to affect about one of every 36 American children. New research has shown that it may be possible to d ...
    Written By: Carmen Leitch
    3
    FEB 29, 2024
    Immunology
    Protein Signal Drives Pro-Tumor Myeloid Genesis
    Protein Signal Drives Pro-Tumor Myeloid Genesis
    Myeloid cells are a population of cells classified to denote a specific lineage. “Myeloid” specifically refe ...
    Written By: Brian G. Morreale
    5
     
     
  • FEB 29, 2024
    Immunology
    Novel Gene Editing Tool Improves Immune Cell Activity
    Novel Gene Editing Tool Improves Immune Cell Activity
    Jennifer Doudna and Emmanuelle Charpentier were awarded the 2020 Nobel Prize in Chemistry for their pioneering work in g ...
    Written By: Brian G. Morreale
    5
    FEB 15, 2024
    Immunology
    New Mechanism Reveals How Tumors Evade Immune Detection
    New Mechanism Reveals How Tumors Evade Immune Detection
    Scientists have dedicated their lives to finding a cure for cancer. America became a science hub for cancer research, pa ...
    Written By: Brian G. Morreale
    4
    FEB 06, 2024
    Genetics & Genomics
    Another CRISPR-Based Therapy Relieves Patients of Disease Symptoms
    Another CRISPR-Based Therapy Relieves Patients of Disease Symptoms
    The CRISPR gene editing tool was developed about a decade ago, and since then it has transformed research in the life sc ...
    Written By: Carmen Leitch
    4
    FEB 01, 2024
    Cancer
    Understanding the Links between Circadian Clock Tumor Growth
    Understanding the Links between Circadian Clock Tumor Growth
    The circadian clock describes the 24-hour internal clock in our brains.  The rhythms that regulate our awake and sl ...
    Written By: Katie Kokolus
    4
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