New Gene Therapy
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- All
- News
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Bad Cholesterol Reduction: Single Gene Therapy Dose Shows 62% Drop
A one-time gene therapy for treating hypercholesterolaemia could lower up to 62 per cent 'bad' cholesterol in the blood, with reductions sustaining over time, according to results of a clinical trial published in The New England Journal of Medicine.
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New One-Shot Gene Editing Therapy May Reduce Bad Cholesterol By 62%
A groundbreaking gene-editing therapy developed by Eli Lilly and Verve Therapeutics reduced "bad" LDL cholesterol by up to 62% after a single infusion in an early trial, raising hopes for a long-lasting treatment to reduce heart attack risk
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Liver Health Breakthrough: New Study Reveals How Gut Bacteria Can Restore Ageing Livers
A new study published in Digestive Disease Week reveals that good gut bacteria could restore ageing liver tissue. Here is what the study found and why it matters for liver health therapies.
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A New Gene Therapy May Restore Hearing In Weeks, Give People Born Deaf A Chance To Hear Again
The treatment targets a specific type of inherited deafness caused by mutations in a gene called OTOF.
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Rare Epilepsy Breakthrough: Why This New Treatment Is Reducing Seizure Frequency In Children By 91%
An international clinical trial has achieved a new effective treatment option for rare epilepsy that can reduce seizures by 91% in children. Here is what this breakthrough means for the children suffering from Dravet Syndrome.
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FDA Push For Fake Brain Surgery Trial Spurs Ethical Concerns
The FDA's request, intended to conclusively determine the therapy's potency, has raised concerns the trial would be both unethical and impractical.
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Top Scientific Discoveries Of 2025: A Year of Quantum Leaps And Cosmic Revelations
Here's a look at scientific discoveries and breakthroughs that made headlines in 2025, spanning space, biology, ecology, and fundamental physics.
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New Research On Gene Editing Therapy May Cut Cholesterol, Triglycerides By Half
Melbourne, Nov 10 (IANS) Researchers in Australia have led a first-in-human trial for a breakthrough gene-editing therapy that halves bad cholesterol and triglycerides in people with difficult-to-treat lipid disorders.
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Scientists Restore Ancient Gene To Help Treat Gout And Uric Acid Disorders
Scientists at Georgia State University have revived a 20-million-year-old gene called uricase, which may offer new hope in treating gout and related uric acid disorders by helping the body break down excess uric acid naturally.
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First Mouse with Two Fathers Thrives into Adulthood, Marking New Scientific Milestone
Scientists have achieved a major milestone by creating a bi-paternal mouse, marking progress in stem cell science and reproductive biology. By modifying imprinting genes, researchers overcame previous barriers to unisexual mammalian reproduction. While only a small percentage of the engineered embryos survived to birth, the research demonstrates th...
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AI Unlocks “DNA Grammar” for Targeted Gene Editing, Enhancing Gene Therapy, and More
Researchers at The Jackson Laboratory, Broad Institute, and Yale University use AI to identify DNA sequences that control gene activation in specific cells. This AI model decodes patterns in DNA regions called cis-regulatory elements (CREs), guiding synthetic DNA switches for cell-specific gene activity. Early testing in animal models showed succes...
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Innovative mRNA Delivery Technique Could Correct Genetic Disorders Before Birth
A recent study from UC Davis and UC Berkeley has unveiled a groundbreaking mRNA delivery method capable of editing genes in fetal brain cells. By administering mRNA encapsulated in lipid nanoparticles, researchers successfully targeted genetic disorders such as Angelman syndrome before birth. This approach, which minimizes risks of inflammation, of...
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New Research Aims To Decode Genetic Roots Of Mental Illness
Neurodevelopmental and psychiatric diseases (NPD), such as schizophrenia, bipolar disorder, autism, and depression, have a negative impact on individuals, families, and society. Unfortunately, effective therapies are often lacking.
-
1-Year-Old UK Girl's Hearing Restored After Groundbreaking Gene Therapy
An 18-month-old British girl who was born completely deaf is believed to be the youngest person to have their hearing restored after undergoing groundbreaking new gene therapy.
-
New Research Suggests Gene Therapy Could Be Used To Treat Pitt-Hopkins Syndrome
Postnatal gene therapy may be able to prevent or repair many of the harmful effects of Pitt-Hopkins syndrome, a rare genetic condition, according to researchers at the University of North Carolina School of Medicine. In an animal model of Pitt-Hopkins syndrome, researchers discovered that restoring lost gene activity eliminates numerous clinical in...
-
Bad Cholesterol Reduction: Single Gene Therapy Dose Shows 62% Drop
A one-time gene therapy for treating hypercholesterolaemia could lower up to 62 per cent 'bad' cholesterol in the blood, with reductions sustaining over time, according to results of a clinical trial published in The New England Journal of Medicine.
-
New One-Shot Gene Editing Therapy May Reduce Bad Cholesterol By 62%
A groundbreaking gene-editing therapy developed by Eli Lilly and Verve Therapeutics reduced "bad" LDL cholesterol by up to 62% after a single infusion in an early trial, raising hopes for a long-lasting treatment to reduce heart attack risk
-
Liver Health Breakthrough: New Study Reveals How Gut Bacteria Can Restore Ageing Livers
A new study published in Digestive Disease Week reveals that good gut bacteria could restore ageing liver tissue. Here is what the study found and why it matters for liver health therapies.
-
A New Gene Therapy May Restore Hearing In Weeks, Give People Born Deaf A Chance To Hear Again
The treatment targets a specific type of inherited deafness caused by mutations in a gene called OTOF.
-
Rare Epilepsy Breakthrough: Why This New Treatment Is Reducing Seizure Frequency In Children By 91%
An international clinical trial has achieved a new effective treatment option for rare epilepsy that can reduce seizures by 91% in children. Here is what this breakthrough means for the children suffering from Dravet Syndrome.
-
FDA Push For Fake Brain Surgery Trial Spurs Ethical Concerns
The FDA's request, intended to conclusively determine the therapy's potency, has raised concerns the trial would be both unethical and impractical.
-
Top Scientific Discoveries Of 2025: A Year of Quantum Leaps And Cosmic Revelations
Here's a look at scientific discoveries and breakthroughs that made headlines in 2025, spanning space, biology, ecology, and fundamental physics.
-
New Research On Gene Editing Therapy May Cut Cholesterol, Triglycerides By Half
Melbourne, Nov 10 (IANS) Researchers in Australia have led a first-in-human trial for a breakthrough gene-editing therapy that halves bad cholesterol and triglycerides in people with difficult-to-treat lipid disorders.
-
Scientists Restore Ancient Gene To Help Treat Gout And Uric Acid Disorders
Scientists at Georgia State University have revived a 20-million-year-old gene called uricase, which may offer new hope in treating gout and related uric acid disorders by helping the body break down excess uric acid naturally.
-
First Mouse with Two Fathers Thrives into Adulthood, Marking New Scientific Milestone
Scientists have achieved a major milestone by creating a bi-paternal mouse, marking progress in stem cell science and reproductive biology. By modifying imprinting genes, researchers overcame previous barriers to unisexual mammalian reproduction. While only a small percentage of the engineered embryos survived to birth, the research demonstrates th...
-
AI Unlocks “DNA Grammar” for Targeted Gene Editing, Enhancing Gene Therapy, and More
Researchers at The Jackson Laboratory, Broad Institute, and Yale University use AI to identify DNA sequences that control gene activation in specific cells. This AI model decodes patterns in DNA regions called cis-regulatory elements (CREs), guiding synthetic DNA switches for cell-specific gene activity. Early testing in animal models showed succes...
-
Innovative mRNA Delivery Technique Could Correct Genetic Disorders Before Birth
A recent study from UC Davis and UC Berkeley has unveiled a groundbreaking mRNA delivery method capable of editing genes in fetal brain cells. By administering mRNA encapsulated in lipid nanoparticles, researchers successfully targeted genetic disorders such as Angelman syndrome before birth. This approach, which minimizes risks of inflammation, of...
-
New Research Aims To Decode Genetic Roots Of Mental Illness
Neurodevelopmental and psychiatric diseases (NPD), such as schizophrenia, bipolar disorder, autism, and depression, have a negative impact on individuals, families, and society. Unfortunately, effective therapies are often lacking.
-
1-Year-Old UK Girl's Hearing Restored After Groundbreaking Gene Therapy
An 18-month-old British girl who was born completely deaf is believed to be the youngest person to have their hearing restored after undergoing groundbreaking new gene therapy.
-
New Research Suggests Gene Therapy Could Be Used To Treat Pitt-Hopkins Syndrome
Postnatal gene therapy may be able to prevent or repair many of the harmful effects of Pitt-Hopkins syndrome, a rare genetic condition, according to researchers at the University of North Carolina School of Medicine. In an animal model of Pitt-Hopkins syndrome, researchers discovered that restoring lost gene activity eliminates numerous clinical in...
