Preclinical Evaluation of the Systemic Safety, Efficacy, and Biodistribution of a Recombinant AAV8 Vector Expressing FIX-TripleL in Hemophilia B Mice: Implications for Human Gene Therapy. [PDF]
Chou SC +11 more
europepmc +1 more source
Intracranial hemorrhage in an infant leads to the diagnosis and treatment of severe hemophilia B: a case report. [PDF]
Lassandro G, Palladino V, Giordano P.
europepmc +1 more source
Plasma levels of Human Granulocytic Elastase-alpha-Proteinase Inhibitor Complex (E-alpha1PI) in Leukemia [PDF]
Hiller, E., Jochum, Marianne
core
CRISPR/Cas9 Edition of the F9 Gene in Human Mesenchymal Stem Cells for Hemophilia B Therapy. [PDF]
Lara-Navarro IJ +3 more
europepmc +1 more source
Universal base editing for hemophilia B [PDF]
Nemekhbayar Baatartsogt +16 more
openalex +1 more source
Retrospective analysis of hemophilia B in Turkey: identifying main characteristics and treatment options. [PDF]
Zülfikar B +41 more
europepmc +1 more source
[Gene therapy marks the beginning of a potential "clinical cure" for hemophilia B patients]. [PDF]
Zhang L.
europepmc +1 more source
AAV vectors for long-term gene therapy of hemophilia B: Are we there yet? [PDF]
Srivastava A.
europepmc +1 more source
Lentiviral-Engineered Mesenchymal Stem Cells for Hemophilia B Gene Therapy
Megan Dodd
openalex +1 more source

