Results 241 to 250 of about 214,719 (303)

Characterization of difficult-to-remove host cell proteins in adeno-associated virus downstream processing. [PDF]

Mol Ther Methods Clin Dev
Leibiger TM, Min L, Lee KH.
europepmc   +1 more source

Gene Editing for Haemophilia—The Next Frontier

Haemophilia, EarlyView.
ABSTRACT The recently approved haemophilia A and B gene therapies via adeno‐associated virus (AAV) showed a promising therapeutic response after a single injection, but there are still limitations, including the potential loss of transgene expression and restriction in adults.
Mirko Pinotti, Gregory A. Newby, Sundar Selvaraj, Steven W. Pipe   +3 more
wiley   +1 more source

Methodological validation of a cryo-TEM-based detection method for empty capsid ratio in recombinant adeno-associated virus. [PDF]

Biochem Biophys Rep
Xu L, Chen J, Zhu W, Zhao Y, Zheng F, Du R, Jiang Y, Yang Y, Chen Y, Dong Y, Zhang Z, Tong Y.   +11 more
europepmc   +1 more source

Final Analysis of the Phase 1/2 Trial of Valoctocogene Roxaparvovec for Severe Haemophilia A

Haemophilia, EarlyView.
ABSTRACT Introduction Valoctocogene roxaparvovec is an adeno‐associated virus vector serotype 5 (AAV5)‐mediated gene therapy for severe haemophilia A (HA). Aim Report the final safety and efficacy results of the phase 1/2 trial of valoctocogene roxaparvovec. Methods An open‐label phase 1/2 trial (NCT02576795) enrolled adult males with severe HA (factor
Priyanka Raheja, Savita Rangarajan, Will Lester, Bella Madan, Glenn F. Pierce, Emily Symington, Carolyn Millar, Dane Osmond, Mingjin Li, Konstantia‐Maria Chavele   +9 more
wiley   +1 more source

Adeno-associated virus and hepatocytes: frenemies? [PDF]

Blood Adv
Pierce GF.
europepmc   +1 more source

Respiratory Viral Infections and the Tonsillar Transcriptome: An Exploratory Study

Clinical &Experimental Allergy, EarlyView.
Tanzeela Hanif, Lotta E. Ivaska, Antti Silvoniemi, Emilia Mikola, Tuomo Puhakka, Oscar Palomares, Sanna Toppila‐Salmi, Mubeccel Akdis, Cezmi A. Akdis, Tuomas Jartti   +9 more
wiley   +1 more source

Seventh Åland Island Meeting on von Willebrand Disease

Haemophilia, EarlyView.
ABSTRACT Introduction The seventh Åland Island Meeting on von Willebrand Disease (VWD) was held on the Åland archipelago in Finland, from 26 to 28 September 2024. Aim The meeting brought together experts in the field of VWD from around the world to share the latest advances and knowledge in VWD.
Riitta Lassila, Ruben Bierings, Nathan T. Connell, Fernando F. Corrales‐Medina, Augusto B. Federici, Veronica H. Flood, Floor Heubel‐Moenen, Jill M. Johnsen, Geoffrey Kuppens, Davide Matino, Carolyn Millar, Ingmar Schoen, Anish Sharda, Robert F. Sidonio Jr, Alok Srivastava, Timea Szanto, Angela C. Weyand, Erik Berntorp   +17 more
wiley   +1 more source

The predawn dilemma in adeno-associated virus-based gene therapies for hereditary deafness. [PDF]

Am J Stem Cells
Zhou J, Deng D, Gan C, Du J, Zhao Y.
europepmc   +1 more source

Defining the Role of Nurses in Gene Therapy for Haemophilia

Haemophilia, EarlyView.
Abstract Introduction Haemophilia is a rare bleeding disorder now undergoing a major shift in care as gene therapy (GT), delivered through viral‑vector in vivo gene transfer, has enabled adults with haemophilia A or B to stop prophylaxis with minimal bleeding.
Greta Mulders, Simon Fletcher, Aysen Tasgin, Marica Moro, Marléne Beijlevelt, Kate Khair   +5 more
wiley   +1 more source

Role of viral protein ratio in the structure and separation of empty and full adeno-associated virus capsids: A molecular dynamics study. [PDF]

Mol Ther Adv
Valiya Parambathu A, Kumar V, Khanal O, Lenhoff AM.   +3 more
europepmc   +1 more source