Results 51 to 60 of about 92,053 (154)
Engineering Biology Beyond Single Genes: Advances and Challenges in Multiplex Genome Editing
Animal Research and One Health, EarlyView.Multiplex genome editing is transforming genome engineering from single‐gene perturbation to network‐level control, yet its broader application remains limited by challenges in gRNA array engineering, delivery technologies, and safety management. Emerging AI‐driven approaches are accelerating guide RNA design and CRISPR effector optimization for ...
Linli Wang, Yongbin Liu, Hongbing Han
wiley +1 more source
British Journal of Clinical Pharmacology, EarlyView.Small and mid‐sized pharmaceutical innovators often have limited in‐house health economics and market access expertise, and may struggle to align development strategies of investigational medicinal products with health system needs and payer expectations.
Zoltán Kaló +5 more
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Cellular and Molecular Life SciencesCystic fibrosis (CF) is a monogenic disorder caused by mutations in the CFTR gene, which encodes a cAMP-regulated anion channel at the apical plasma membrane (PM) of epithelial cells. CFTR modulators have recently been approved as effective therapies for
Hazuki Tateishi +3 more
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CFTR Modulator Response in Nasal Organoids Derived from People with Cystic Fibrosis
CellsDespite the progressive extension of CFTR variant eligibility to the triple combination of elexacaftor/tezacaftor/ivacaftor (ETI), most rare CFTR pathogenic variants remain ineligible for CFTR modulators.
Stefania Lo Cicero +15 more
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Modulators of CFTR protein function in the treatment of cystic fibrosis - a literature review
Journal of Education, Health and Sport, 2022 Cystic fibrosis (CF) is a multi-system genetic disease with an autosomal recessive inheritance mechanism. The breakthroughs in the therapy of patients with CF turned out to be modulators of CFTR protein function.
Dariusz Chojęta +6 more
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Precision medicine in paediatrics: Progress and priorities
British Journal of Clinical Pharmacology, EarlyView.Precision medicine is revolutionizing personalized healthcare, advancing both diagnostics and therapeutics at an unprecedented pace. Reviewing the paediatric applications of pharmacometrics, pharmacogenomics and advanced therapy medicinal products highlights not only the relevance of these exciting innovations to frontline care but also the significant
Nicola Husain +3 more
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ChildrenBackground/Objectives: Cystic fibrosis (CF) is a multisystemic and genetic disorder. Mutations in the CF transmembrane conductance regulator (CFTR) gene impair the function of the CFTR protein, leading to various complications in multiple organs, mainly ...
Katerina Iordanidou +6 more
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Status and future of recombinant adeno‐associated virus vector manufacturing
Biotechnology Progress, EarlyView.Abstract Sixty years of adeno‐associated virus (AAV) research illustrates a trajectory marked by basic science exploration, iterative innovation, persistent challenges, a number of clinical setbacks, as well as commercial therapeutic triumphs. This continual evolution has led to recombinant AAV (rAAV) becoming a cornerstone of modern gene therapy ...
Frank Agbogbo, David Dismuke
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ERJ Open Research, 2019 Cystic fibrosis (CF) is caused by mutations in the CF transmembrane conductance regulator gene (CFTR) that result in diminished quantity and/or function of the CFTR anion channel.
Jennifer L. Taylor-Cousar +11 more
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JPGN Reports, EarlyView.Abstract Objectives The efficacy of cystic fibrosis transmembrane conductance regulator (CFTR)‐modulator therapies in preventing or ameliorating cystic fibrosis liver disease (CFLD) by correcting CFTR in cholangiocytes is not well‐documented. This study aimed to assess liver function during CFTR‐modulators.
Laura Giugliano +12 more
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