Genetic 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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Nick Jonas' Friend Maya Kibbel Dies Of Wilson's Disease At 30: Signs And Risks Of The Rare Disease Explained
Nick Jonas lost his friend Maya Kibbel to Wilson's disease. Here is how the rare genetic disorder can manifest in the human body, along with the available treatment options.
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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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Viruses and Bacteria Evolve Differently in Space, ISS Study Finds
Viruses and bacteria evolved differently aboard the ISS.Microgravity reshaped infection behavior and genetic mutations. Research may advance phage therapy and combat drug resistance.
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CRISPR Explained: How Gene Editing Is Reshaping Medicine
CRISPR is revolutionizing medicine by enabling precise DNA edits, treating genetic diseases like sickle cell anemia and beta-thalassemia, and pioneering in vivo editing and personalized therapies.
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US Woman With Stage 4, 'Incurable' Colon Cancer Saved By Breakthrough Treatment
Diagnosed with Stage 4 colon cancer, Ms Dimery underwent chemotherapy, radiation and other combination therapies -- none of which seemingly worked.
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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.
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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.
-
Nick Jonas' Friend Maya Kibbel Dies Of Wilson's Disease At 30: Signs And Risks Of The Rare Disease Explained
Nick Jonas lost his friend Maya Kibbel to Wilson's disease. Here is how the rare genetic disorder can manifest in the human body, along with the available treatment options.
-
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.
-
Viruses and Bacteria Evolve Differently in Space, ISS Study Finds
Viruses and bacteria evolved differently aboard the ISS.Microgravity reshaped infection behavior and genetic mutations. Research may advance phage therapy and combat drug resistance.
-
CRISPR Explained: How Gene Editing Is Reshaping Medicine
CRISPR is revolutionizing medicine by enabling precise DNA edits, treating genetic diseases like sickle cell anemia and beta-thalassemia, and pioneering in vivo editing and personalized therapies.
-
US Woman With Stage 4, 'Incurable' Colon Cancer Saved By Breakthrough Treatment
Diagnosed with Stage 4 colon cancer, Ms Dimery underwent chemotherapy, radiation and other combination therapies -- none of which seemingly worked.
-
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.
