Status and future of recombinant adeno‐associated virus vector manufacturing
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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[Multiple clinical effects of recombinant human growth hormone therapy]. [PDF]
Zhou B, Yang Y.
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ABSTRACT We here describe mouse models with complementary homozygous Svil mutations. In skeletal muscle, Svil‐Mut mice express the Svil‐encoded N‐terminus fused to the βgal‐neo gene‐trap tag and lack the highly conserved archvillin C‐terminus; Svil‐KO mice lack expression of all known Svil‐encoded proteins; and Svil‐LoxP mice contain loxP sites for ...
Tara C. Smith +9 more
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Case Report: Clinical phenotypes and recombinant human growth hormone therapeutic exploration for a patient with Takenouchi-Kosaki syndrome harboring the <i>CDC42</i> p.Arg68Gln variant. [PDF]
Lin XX, Song YY, Xue JC, Liu H.
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Long‐Acting Therapeutics in Pediatric Health: Bridging Innovation and Access
Children have distinct therapeutic needs arising from age‐dependent physiology, disease epidemiology, formulation requirements, dosing considerations, and safety vulnerabilities. Despite substantial reductions in childhood mortality over the past three decades, nearly 5 million children under five died in 2023, with infectious diseases continuing to ...
Prajith Venkatasubramanian +5 more
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Efficacy and influencing factors of recombinant human growth hormone therapy in children with Turner syndrome: a single-center retrospective cohort study. [PDF]
Chen J +9 more
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Role of selenium in the pathophysiology of cardiorenal anaemia syndrome
Abstract Chronic kidney disease (CKD) and cardiovascular disease (CVD) have multiple bidirectional mechanisms, and anaemia is one of the critical factors that are associated with the progression of the two disorders [referred to as cardiorenal anaemia syndrome (CRAS)].
Shigeyuki Arai +2 more
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KBG Syndrome: A Case Report and Longitudinal Assessment of Long-Acting Recombinant Human Growth Hormone Therapy. [PDF]
Nan H, Zhang P, Qian J.
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Systemic aging fuels heart failure: Molecular mechanisms and therapeutic avenues
Abstract Systemic aging influences various physiological processes and contributes to structural and functional decline in cardiac tissue. These alterations include an increased incidence of left ventricular hypertrophy, a decline in left ventricular diastolic function, left atrial dilation, atrial fibrillation, myocardial fibrosis and cardiac ...
Zhuyubing Fang +7 more
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Metabolic and bone effects of long-acting polyethylene glycol-recombinant human growth hormone in growth hormone deficiency during the transition period: a prospective study. [PDF]
Huang K +8 more
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