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Discovery enables effective use of gene therapy for muscular dystrophies and other large-gene diseases

Discovery enables effective use of gene therapy for muscular dystrophies and other large-gene diseases

byUniversity of Rochester Medical CenterCredit: Pixabay/CC0 Public DomainGene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there

Discovery enables effective use of gene therapy for muscular dystrophies and other large-gene diseases

byUniversity of Rochester Medical CenterCredit: Pixabay/CC0 Public DomainGene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there
Huntington's disease gene may also enhance early brain development and intelligence

Huntington's disease gene may also enhance early brain development and intelligence

by Jennifer Brown,University of IowaYears-to-onset (YTO)-dependent brain functional changes. Credit:Annals of Neurology(2024). DOI: 10.1002/ana.27046The genetic mutation that causes

Huntington's disease gene may also enhance early brain development and intelligence

by Jennifer Brown,University of IowaYears-to-onset (YTO)-dependent brain functional changes. Credit:Annals of Neurology(2024). DOI: 10.1002/ana.27046The genetic mutation that causes
A new strategy to enhance gene therapy for sickle cell disease

A new strategy to enhance gene therapy for sickle cell disease

byMary Ann Liebert, IncCyclosporin H Improves the Transduction of CD34+ Cells with an Anti-Sickling Globin Vector, a Possible Therapeutic Approach for Sickle Cell Disease. Credit: Mary Ann Liebe

A new strategy to enhance gene therapy for sickle cell disease

byMary Ann Liebert, IncCyclosporin H Improves the Transduction of CD34+ Cells with an Anti-Sickling Globin Vector, a Possible Therapeutic Approach for Sickle Cell Disease. Credit: Mary Ann Liebe
Discovery of new genes for molar pregnancies sheds light on their increased incidence in women aged 35 and over

Discovery of new genes for molar pregnancies sheds light on their increased incidence in women aged 35 and over

byMcGill UniversityGraphical abstract. Credit:Journal of Clinical Investigation(2024). DOI: 10.1172/JCI170669A molar pregnancy, also known as a hydatidiform mole, is an abnormal huma

Discovery of new genes for molar pregnancies sheds light on their increased incidence in women aged 35 and over

byMcGill UniversityGraphical abstract. Credit:Journal of Clinical Investigation(2024). DOI: 10.1172/JCI170669A molar pregnancy, also known as a hydatidiform mole, is an abnormal huma
Scientists discover a rare missense variant in STAT6 that protects against asthma

Scientists discover a rare missense variant in STAT6 that protects against asthma

by deCODE geneticsp.L406P is a partial LOF variant. Credit:Journal of Allergy and Clinical Immunology(2024). DOI: 10.1016/j.jaci.2024.10.002Scientists at deCODE genetics and collaborators

Scientists discover a rare missense variant in STAT6 that protects against asthma

by deCODE geneticsp.L406P is a partial LOF variant. Credit:Journal of Allergy and Clinical Immunology(2024). DOI: 10.1016/j.jaci.2024.10.002Scientists at deCODE genetics and collaborators
Genetic platform identifies 'Achilles heel' of drug-resistant Mycobacterium tuberculosis

Genetic platform identifies 'Achilles heel' of drug-resistant Mycobacterium tuberculosis

byUniversity of OtagoWG-CRISPRi screening in M. tuberculosis strain mc26206. Credit:Nature Communications(2024). DOI: 10.1038/s41467-024-54072-wA University of Otago-led study has fo

Genetic platform identifies 'Achilles heel' of drug-resistant Mycobacterium tuberculosis

byUniversity of OtagoWG-CRISPRi screening in M. tuberculosis strain mc26206. Credit:Nature Communications(2024). DOI: 10.1038/s41467-024-54072-wA University of Otago-led study has fo
Promising results from phase 1 gene therapy trial for Danon disease

Promising results from phase 1 gene therapy trial for Danon disease

byChildren's Hospital of PhiladelphiaCredit: Pixabay/CC0 Public DomainResearchers at Children's Hospital of Philadelphia (CHOP) announced encouraging results from the first ever gene the

Promising results from phase 1 gene therapy trial for Danon disease

byChildren's Hospital of PhiladelphiaCredit: Pixabay/CC0 Public DomainResearchers at Children's Hospital of Philadelphia (CHOP) announced encouraging results from the first ever gene the
Genetic study links heart shape to cardiovascular disease

Genetic study links heart shape to cardiovascular disease

byQueen Mary, University of LondonHeart shape component summary. Credit:Nature Communications(2024). DOI: 10.1038/s41467-024-53594-7A new, multinational study has revealed that the s

Genetic study links heart shape to cardiovascular disease

byQueen Mary, University of LondonHeart shape component summary. Credit:Nature Communications(2024). DOI: 10.1038/s41467-024-53594-7A new, multinational study has revealed that the s
Clinical trial finds new gene editing therapy for heart condition to be safe and effective

Clinical trial finds new gene editing therapy for heart condition to be safe and effective

byUniversity College LondonCredit: Pixabay/CC0 Public DomainA new type of therapy that "edits" a gene in patients with a rare heart condition has been shown to be safe and effective, a

Clinical trial finds new gene editing therapy for heart condition to be safe and effective

byUniversity College LondonCredit: Pixabay/CC0 Public DomainA new type of therapy that "edits" a gene in patients with a rare heart condition has been shown to be safe and effective, a
New resource maps gene-disease links across common conditions, paving the way for personalized medicine

New resource maps gene-disease links across common conditions, paving the way for personalized medicine

byHebrew University of JerusalemCredit: Pixabay/CC0 Public DomainA new study introduces an innovative tool for exploring gene-disease connections: the PWAS Hub. This resource is based on the nov

New resource maps gene-disease links across common conditions, paving the way for personalized medicine

byHebrew University of JerusalemCredit: Pixabay/CC0 Public DomainA new study introduces an innovative tool for exploring gene-disease connections: the PWAS Hub. This resource is based on the nov
Molecular detection method tracks outbreak of drug-resistant fungi

Molecular detection method tracks outbreak of drug-resistant fungi

by Juliane Seeber,Friedrich Schiller University of JenaTimeline and hospital network dynamics of a Candida parapsilosis outbreak in Berlin, Germany. Credit:The Lancet Microbe(2024).

Molecular detection method tracks outbreak of drug-resistant fungi

by Juliane Seeber,Friedrich Schiller University of JenaTimeline and hospital network dynamics of a Candida parapsilosis outbreak in Berlin, Germany. Credit:The Lancet Microbe(2024).
New drug targets for Alzheimer's identified from cerebrospinal fluid

New drug targets for Alzheimer's identified from cerebrospinal fluid

by Mark Reynolds,Washington University School of MedicineCellular localization of immune and lysosomal proteins. Credit:Nature Genetics(2024). DOI: 10.1038/s41588-024-01972-8A multit

New drug targets for Alzheimer's identified from cerebrospinal fluid

by Mark Reynolds,Washington University School of MedicineCellular localization of immune and lysosomal proteins. Credit:Nature Genetics(2024). DOI: 10.1038/s41588-024-01972-8A multit