Results 71 to 80 of about 2,418 (178)
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
wiley +1 more source
Cystic fibrosis, molecular genetics for all life
Journal of Pediatric and Neonatal Individualized Medicine, 2015 Cystic fibrosis (CF) is the most frequent lethal autosomal recessive disorder among Caucasians (incidence: 1:2,500 newborn). In the last two decades CF prognosis considerably improved and many patients well survive into their adulthood.
Ausilia Elce +5 more
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Clinical Pharmacology &Therapeutics, EarlyView.Precision medicine offers the opportunity to improve the benefit–risk profile of new therapies by prospectively identifying patients most likely to respond or least likely to experience harm; however, its systematic integration into drug development remains inconsistent outside oncology.
Ingrid Holst‐Laubjerg +1 more
wiley +1 more source
CFTR Functions as a Tumor Suppressor and Is Regulated by DNA Methylation in Colorectal Cancer
Cancer Management and Research, 2020 Can Liu,1 Chao Song,2 Jiaxi Li,3 Qing Sun1,3 1Department of Pathology, Shandong Provincial Qianfoshan Hospital, Cheeloo College of Medicine, Shandong University, Jinan, Shandong Province, People’s Republic of China; 2Department of Pathology, Zibo ...
Liu C, Song C, Li J, Sun Q
doaj
Epilepsia, EarlyView.Abstract Absence seizures evolving to bilateral tonic–clonic seizures may have focal or asymmetric clinical features. Using quantitative electroencephalography (EEG) may evaluate this phenomenon and the involved neuronal network. We conducted a retrospective chart review of pediatric patients with generalized epilepsy and normal brain magnetic ...
Juan Toro‐Perez +8 more
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Cellular and Molecular Gastroenterology and Hepatology, 2020 Backgraoud & Aims: Aberrant epithelial bicarbonate (HCO3−) secretion caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene is associated with several diseases including cystic fibrosis and pancreatitis.
Yonjung Kim +10 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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Efficacy and Mechanism Evaluation, 2016 Background: Cystic fibrosis (CF) is a chronic, life-limiting disease caused by mutations in the CF transmembrane conductance regulator (CFTR) gene leading to abnormal airway surface ion transport, chronic lung infections, inflammation and eventual ...
Eric WFW Alton +80 more
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Neuroprotection, EarlyView.Abstract Prime editing, a novel clustered regularly interspaced short palindromic repeats (CRISPR)‐based technology, fuses a reverse transcriptase (RT) to an engineered CRISPR‐associated protein 9 (Cas9) and uses a prime editing guide RNA (pegRNA)‐encoded template.
Tianshan Ji +4 more
wiley +1 more source
Current Cell/Organoid and Animal Models for Primary Sclerosing Cholangitis
Portal Hypertension &Cirrhosis, EarlyView.Primary sclerosing cholangitis (PSC) is a chronic cholestatic liver disease with limited therapeutic options and a marked risk of progression to biliary fibrosis, cirrhosis, and malignancy. Progress in PSC research has been hindered by the lack of models that faithfully recapitulate the complex biliary microenvironment and disease heterogeneity ...
Qigu Yao +4 more
wiley +1 more source