INDUCTION OF TOLERANCE BY ORAL ADMINISTRATION OF FACTOR VIII AND TREATMENT OF HEMOPHILA [PDF]
Disclosed herein is a simple method for the treatment of antigen-deficiency diseases, by orally administering to a Subject a therapeutically effective amount of the deficient anti gen, wherein the antigen is not present in a liposome.
Alpan, Oral +3 more
core +1 more source
Multivariable model integrating PHI and mpMRI for detecting csPCa in biopsy‐naïve men
Abstract Background The integration of blood‐based biomarkers and multiparametric magnetic resonance imaging (mpMRI) has been proposed to improve prostate cancer (PCa) diagnosis. However, few validated models combine both tools to support risk‐adapted clinical decision‐making.
Mario Dominguez Esteban +13 more
wiley +1 more source
Persistent Expression of hF.IX After Tolerance Induction by In Utero or Neonatal Administration of AAV-1-F.IX in Hemophilia B Mice [PDF]
Denise E. Sabatino +7 more
openalex +1 more source
Von Drygalski A, Giermasz A, Castaman G, et al. Etranacogene dezaparvovec (AMT-061 phase 2b): normal/near normal FIX activity and bleed cessation in hemophilia B. Blood Adv. 2019;3(21):3241-3247. [PDF]
openalex +1 more source
ABSTRACT This paper proposes the controlled release of adeno‐associated virus (AAV) by polymer mesh structures of alginate hydrogel microbeads (gel‐beads). The polymer mesh structure of alginate hydrogel can be controlled by adjusting the number of cross‐linking points depending on the molecular structure (M/G ratio) of the alginate hydrogel.
Aiki Hioki +3 more
wiley +1 more source
Clinical audit of inherited bleeding disorders in a developing country
Objective: We did a clinical audit to determine the status of coagulation disorders in a hemophilia care center in Pakistan. Setting: Fatimid foundation blood bank and hematological diseases center, Lahore.
Sajid Raihan +4 more
doaj
cGMP Recombinant FIX for IV and Oral Hemophilia B Therapy [PDF]
Three specific aims are proposed: Specific Aim # 1. Process engineer and scale-up the recovery and purification of transgenic recombinant human Factor IX.
Dernell, William, KEY PERSON +9 more
core +1 more source
Enhanced pharmacokinetic properties of a glycoPEGylated recombinant factor IX: a first human dose trial in patients with hemophilia B [PDF]
Claude Négrier +4 more
openalex +1 more source
Model‐Informed Drug Development Applications and Opportunities in mRNA‐LNP Therapeutics
The utilization of lipid nanoparticles (LNP) for encapsulating mRNA has revolutionized the field of therapeutics, enabling the rapid development of COVID‐19 vaccines and cancer vaccines. However, the clinical development of mRNA‐LNP therapeutics faces numerous challenges due to their complex mechanisms of action and limited clinical experience.
Jiawei Zhou +6 more
wiley +1 more source

