Multiple intracerebral hemorrhagic lesions complicating minimal cranioencephalic trauma in a child with severe hemophilia A, trisomy 21 and sickle cell trait carrier: a case report PDF TOURE SokhnaTelemedicine: the future of Humanitarian health sector PDF Lodi Chiara...
Hemophilia B is a hereditary bleeding disorder caused by the deficiency in coagulation factor IX. Understanding coagulation and the role of factor IX as well as patient population and diagnosis are all critical factors in developing treatment strategies and regimens for hemophilia B patients. Current ...
Patients grappling with HCC face an urgent demand for novel therapeutic approaches, and GT emerges as a captivating avenue, holding substantial potential primarily attributed to the distinctive circulatory arrangement of the liver. Notably, the liver predominantly relies on the portal vein for the bulk ...
multifunctional monomer may play a key for future modification. In general, the majority of investigations focus on gene delivery in vitro and in vivo, lacking systematic clinical application. Therefore, based on the existing research foundation, systematic clinical trial studies are imperative for the ...
Pontus A Bostrm,JM Fernández-Real,C Mantzoros - 《Metabolism Clinical & Experimental》 被引量: 76发表: 2013年 Advances in Hemophilia: Experimental Aspects and Therapy This article describes recent clinical and research advances in hemophilia therapy. Different prophylactic regimens for the management ...
Doering CB, Denning G and Shields JE 2018 Preclinical development of a hematopoietic stem and progenitor cell bioengineered factor VIII lentiviral vector gene therapy for hemophilia A. Hum. Gene Ther. 29 1183–1201 Article CAS PubMed PubMed Central Google Scholar Dong W and Kantor B 2021 Lentiv...
Should long-term stability (LTS) studies of controls for ADA assays be performed as they are for PK assays? Monitoring biologically active biomarkers & evaluation of neutralizing antibody via neutralization of this activity Should a pharmacodynamic (PD) readout be considered to better reflect in ...
Omega Therapeutics MORE ON THIS TOPIC Antibody drug conjugate Roche Kicks Off 2025 Committing a Potential $1B for Another ADC From China January 2, 2025 · 2 min read · Annalee Armstrong Gene therapy Sangamo’s Stock Plummets as Pfizer Axes Hemophilia Gene Therapy...
Successful transduction of liver in hemophilia by AAV-factor IX and limitations imposed by the host immune response. Nat Med. 2006;12:342–7. Article CAS Google Scholar Kabanov AV, Kabanov VA. DNA complexes with polycations for the delivery of genetic material into cells. Bioconjug Chem. 1995...
Lentiviral vectors (LVs), derived from human immunodeficiency virus, are powerful tools for modifying the genes of eukaryotic cells such as hematopoietic stem cells and neural cells. With the extensive and in-depth studies on this gene therapy vehicle over the past two decades, LVs have been wide...