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Drug development advances in beneficial loss-of-function mutation targets validated by human genetics

Drug development advances in beneficial loss-of-function mutation targets validated by human genetics

by Sichuan International Medical Exchange and Promotion AssociationTargets primarily include PCSK9, ANGPTL3, HSD17B13, KHK, ASGR1, GPR75, CIDEB, and INHBE. Drug types primarily include small molecules

Drug development advances in beneficial loss-of-function mutation targets validated by human genetics

by Sichuan International Medical Exchange and Promotion AssociationTargets primarily include PCSK9, ANGPTL3, HSD17B13, KHK, ASGR1, GPR75, CIDEB, and INHBE. Drug types primarily include small molecules
Gene therapy gets a turbo boost from researchers

Gene therapy gets a turbo boost from researchers

byUniversity of Hawaii at ManoaGraphical abstract. Credit:Nucleic Acids Research(2024). DOI: 10.1093/nar/gkae534For decades, scientists have dreamed of a future where genetic disease

Gene therapy gets a turbo boost from researchers

byUniversity of Hawaii at ManoaGraphical abstract. Credit:Nucleic Acids Research(2024). DOI: 10.1093/nar/gkae534For decades, scientists have dreamed of a future where genetic disease
Prolonged use of certain hormone drugs linked to increased brain tumor risk

Prolonged use of certain hormone drugs linked to increased brain tumor risk

by British Medical JournalMeningiomas are graded by microscopic appearance, rate of growth and tendency to invade other tissues. Credit: National Cancer InstituteProlonged use of certain progesto

Prolonged use of certain hormone drugs linked to increased brain tumor risk

by British Medical JournalMeningiomas are graded by microscopic appearance, rate of growth and tendency to invade other tissues. Credit: National Cancer InstituteProlonged use of certain progesto
Researchers develop framework to identify molecules responsible for chemotherapy resistance

Researchers develop framework to identify molecules responsible for chemotherapy resistance

JANUARY 18, 2024byKarolinska InstitutetCredit:iScience(2024). https://www.cell.com/iscience/fulltext/S2589-0042(24)00128-7Researchers at Karolinska Institutet and SciLifeLab demonstr

Researchers develop framework to identify molecules responsible for chemotherapy resistance

JANUARY 18, 2024byKarolinska InstitutetCredit:iScience(2024). https://www.cell.com/iscience/fulltext/S2589-0042(24)00128-7Researchers at Karolinska Institutet and SciLifeLab demonstr
Researchers uncover unexpected molecular pattern in fragile X syndrome

Researchers uncover unexpected molecular pattern in fragile X syndrome

byPerelman School of Medicine at the University of PennsylvaniaCredit:Cell(2023). DOI: 10.1016/j.cell.2023.11.019Researchers have found new disrupted genes and an unexpected molecula

Researchers uncover unexpected molecular pattern in fragile X syndrome

byPerelman School of Medicine at the University of PennsylvaniaCredit:Cell(2023). DOI: 10.1016/j.cell.2023.11.019Researchers have found new disrupted genes and an unexpected molecula
CRISPR-based mapping uncovers 'switches' for immune genes central to health

CRISPR-based mapping uncovers 'switches' for immune genes central to health

byGladstone InstitutesTiling CRISPRi screens map gene-specific CREs across the costimulatory locus. Credit:Nature Genetics(2024). DOI: 10.1038/s41588-024-01743-5Healthy T cells are t

CRISPR-based mapping uncovers 'switches' for immune genes central to health

byGladstone InstitutesTiling CRISPRi screens map gene-specific CREs across the costimulatory locus. Credit:Nature Genetics(2024). DOI: 10.1038/s41588-024-01743-5Healthy T cells are t
Scientists develop novel plug-and-play test to evaluate T cell immunotherapy effectiveness

Scientists develop novel plug-and-play test to evaluate T cell immunotherapy effectiveness

byDuke-NUS Medical SchoolT cells are removed from the patient's body, engineered to fight disease, then infused back into the patient's bloodstream. At the point of infusion, and at vari

Scientists develop novel plug-and-play test to evaluate T cell immunotherapy effectiveness

byDuke-NUS Medical SchoolT cells are removed from the patient's body, engineered to fight disease, then infused back into the patient's bloodstream. At the point of infusion, and at vari
Positive parenting strategies can reduce child maltreatment

Positive parenting strategies can reduce child maltreatment

byUniversity of QueenslandOrganization engagement by local government area. Credit:Child Maltreatment(2024). DOI: 10.1177/10775595241259994University of Queensland research shows chi

Positive parenting strategies can reduce child maltreatment

byUniversity of QueenslandOrganization engagement by local government area. Credit:Child Maltreatment(2024). DOI: 10.1177/10775595241259994University of Queensland research shows chi
Study confirms how RNA chemical modifications benefit HIV-1

Study confirms how RNA chemical modifications benefit HIV-1

by Emily Caldwell,The Ohio State UniversityHIV-1 Virus. Credit: J Roberto Trujillo/WikipediaA chemical modification in the HIV-1 RNA genome whose function has been a matter of scientific debate

Study confirms how RNA chemical modifications benefit HIV-1

by Emily Caldwell,The Ohio State UniversityHIV-1 Virus. Credit: J Roberto Trujillo/WikipediaA chemical modification in the HIV-1 RNA genome whose function has been a matter of scientific debate
The double-edge sword of CRISPR application for in vivo studies

The double-edge sword of CRISPR application for in vivo studies

by Impact Journals LLCWorkflow illustration. Credit:Oncotarget(2023). DOI: 10.18632/oncotarget.28459A new editorial paper titled "The double-edge sword of CRISPR application for in vi

The double-edge sword of CRISPR application for in vivo studies

by Impact Journals LLCWorkflow illustration. Credit:Oncotarget(2023). DOI: 10.18632/oncotarget.28459A new editorial paper titled "The double-edge sword of CRISPR application for in vi
These programs put unused prescription drugs in the hands of patients in need

These programs put unused prescription drugs in the hands of patients in need

by Kate Ruder, KFF Health NewsCredit: Pixabay/CC0 Public DomainOn a recent November evening, Angie Phoenix waited at a pharmacy here in Colorado's second-largest city to pick up prescription drugs

These programs put unused prescription drugs in the hands of patients in need

by Kate Ruder, KFF Health NewsCredit: Pixabay/CC0 Public DomainOn a recent November evening, Angie Phoenix waited at a pharmacy here in Colorado's second-largest city to pick up prescription drugs
A new vision for AAV-delivered gene therapies

A new vision for AAV-delivered gene therapies

by Benjamin Boettner,Harvard UniversityAAVs with TLR9-inhibitory sequences incorporated into their genomes allowed significantly higher expression of a linked fluorescent reporter gene in the mo

A new vision for AAV-delivered gene therapies

by Benjamin Boettner,Harvard UniversityAAVs with TLR9-inhibitory sequences incorporated into their genomes allowed significantly higher expression of a linked fluorescent reporter gene in the mo