Results 1 to 10 of about 622,843 (190)

Non-genotoxic conditioning facilitates hematopoietic stem cell gene therapy for hemophilia A using bioengineered factor VIII

Molecular Therapy: Methods & Clinical Development, 2021
Hematopoietic stem and progenitor cell (HSPC) lentiviral gene therapy is a promising strategy toward a lifelong cure for hemophilia A (HA). The primary risks associated with this approach center on the requirement for pre-transplantation conditioning ...
Athena L. Russell, Chengyu Prince, Taran S. Lundgren, Kristopher A. Knight, Gabriela Denning, Jordan S. Alexander, Jaquelyn T. Zoine, H. Trent Spencer, Shanmuganathan Chandrakasan, Christopher B. Doering   +9 more
doaj   +1 more source

Novel plasma exosome biomarkers for prostate cancer progression in co-morbid metabolic disease

Advances in Cancer Biology - Metastasis, 2022
Comorbid Type 2 diabetes (T2D), a metabolic complication of obesity, associates with worse cancer outcomes for prostate, breast, head and neck, colorectal and several other solid tumors.
Naser Jafari, Andrew Chen, Manohar Kolla, Isabella R. Pompa, Yuhan Qiu, Rebecca Yu, Pablo Llevenes, Christina S. Ennis, Joakin Mori, Kiana Mahdaviani, Meredith Halpin, Gretchen A. Gignac, Christopher M. Heaphy, Stefano Monti, Gerald V. Denis   +14 more
doaj   +1 more source

A potential gain-of-function variant of SLC9A6 leads to endosomal alkalinization and neuronal atrophy associated with Christianson Syndrome

Neurobiology of Disease, 2019
Loss-of-function mutations in the recycling endosomal (Na+,K+)/H+ exchanger gene SLC9A6/NHE6 result in overacidification and dysfunction of endosomal-lysosomal compartments, and cause a neurodevelopmental and degenerative form of X-linked intellectual ...
Alina Ilie, Andy Y.L. Gao, Annie Boucher, Jaeok Park, Albert M. Berghuis, Mariëtte J.V. Hoffer, Yvonne Hilhorst-Hofstee, R. Anne McKinney, John Orlowski   +8 more
doaj   +1 more source

Adipocyte-derived shed Syndecan-4 suppresses lipolysis contributing to impaired adipose tissue browning and adaptive thermogenesis

Molecular Metabolism
Lipolysis in white adipose tissue (WAT) provides fatty acids as energy substrates for thermogenesis to increase energy expenditure. Syndecan-4 (Sdc4) is a transmembrane proteoglycan bearing heparan sulfate chains. Although single nucleotide polymorphisms
Jiuyu Zong, Xiaoping Wu, Xiaowen Huang, Lufengzi Yuan, Kai Yuan, Zixuan Zhang, Mengxue Jiang, Zhihui Ping, Lai Yee Cheong, Aimin Xu, Ruby Lai Chong Hoo   +10 more
doaj   +1 more source

Pediatric post-discharge mortality in resource-poor countries: a systematic review and meta-analysisResearch in context

EClinicalMedicine
Summary: Background: Under-five mortality remains concentrated in resource-poor countries. Post-discharge mortality is becoming increasingly recognized as a significant contributor to overall child mortality.
Martina Knappett, Vuong Nguyen, Maryum Chaudhry, Jessica Trawin, Jerome Kabakyenga, Elias Kumbakumba, Shevin T. Jacob, J. Mark Ansermino, Niranjan Kissoon, Nathan Kenya Mugisha, Matthew O. Wiens   +10 more
doaj   +1 more source

Design of the First in Human Gene Therapy Trial of TLT-101 for Chronic Heart Failure (FIGHT-HF)

JACC: Basic to Translational Science
Pia Balmaceda, MD, Tara C. Hitzeman, MPH, Kikuyo E. Shaw, BS, Lynn M. Kolhepp, BS, Matthew Killeen, PhD, James C. Fang, MD, Javed Butler, MD, Adrian F. Hernandez, MD, TingTing Hong, MD, PhD, Robin M. Shaw, MD, PhD   +9 more
doaj   +1 more source

Author Correction: Folding-mediated secretion of pure bispecific antibodies. [PDF]

Nat Biotechnol
Tilegenova C, Liu T, Zhao Q, Are M, Zhao Y, Choi WS, Bhaumik A, Steele R, Manieri NA, Turegun B, Ni A, Cardoso RMF, Shaffer P, Clark D, Ernst R, Li W, Taylor T, Swaminathan SK, Ramaraju B, Liaw K, Jacobs SA, Sharma S, Cheung WC, Zwolak A.   +23 more
europepmc   +1 more source

Impact of CYP2C19 and CYP3A4 Inhibitor Use on Clopidogrel Clinical Effectiveness in CYP2C19 Genotyped Patients Undergoing Percutaneous Coronary Intervention. [PDF]

Clin Pharmacol Ther
Shao D, Malavé JG, Rossi JS, Franchi F, Keeley EC, Angiolillo DJ, Stouffer GA, Cavallari LH, Lee CR.   +8 more
europepmc   +1 more source

Benchmark of Open-Access Star-Allele Callers to Accurately Assess Haplotypes and Phenotypes in Pharmacogenetic Studies. [PDF]

Clin Pharmacol Ther
Gros-La-Faige MB, Génin E, Herzig AF.
europepmc   +1 more source

An intrinsic sequence-structural profile for mRNA-delivered therapeutic antibodies. [PDF]

Brief Bioinform
Mahmoudinobar F, Meng G, Davis JW, Kumar S.   +3 more
europepmc   +1 more source