Results 231 to 240 of about 110,545 (302)

Development of an adeno-associated virus vector for gene replacement therapy of NF1-related tumors. [PDF]

Nat Commun
Bai RY, Shi J, Liu J, Sun N, Lu Y, Chen X, Xu M, Lim H, Li Y, Xu H, Weisgerber K, Ren Z, Pratilas CA, Blakeley JO, Fishel ML, Carrió M, Serra E, Staedtke V.   +17 more
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

pH-dependent DNA degradation pathways for adeno-associated virus gene therapy. [PDF]

Mol Ther Methods Clin Dev
Plegaria JS, Malecka KA, Lin Q, Vogt A, Ausman K, Lopez De Leon A, Petroff M, Barbacci D.   +7 more
europepmc   +1 more source

Treatment With Valoctocogene Roxaparvovec in a Patient With Severe Hemophilia A Led to Sustained Normal FVIII Levels

Haemophilia, EarlyView.
ABSTRACT Introduction Valoctocogene roxaparvovec, an adeno‐associated virus (AAV)‐based gene therapy, enables endogenous factor VIII (FVIII) expression in patients with severe hemophilia A without the need for regular FVIII infusions. Long‐term follow‐up assesses durability, safety, and immune‐related challenges following gene therapy.
Kerstin Herbst, Behnaz Pezeshkpoor, Claudia Klein, Thilo Albert, Philipp Lutz, Christian Strassburg, Ulrich Spengler, Georg Goldmann, Johannes Oldenburg   +8 more
wiley   +1 more source

Novel Human Heart-Derived Natural Adeno-Associated Virus Capsid Combines Cardiospecificity With Cardiotropism In Vivo. [PDF]

Circulation
Zeissler D, Busch M, Grieskamp S, Meinhardt E, Wurzer H, Kulkarni SR, Schrattel AK, Molter S, Kehr D, Qatato M, Rummel C, Hansen A, Loos M, Wrona KM, Völkers M, Kmietczyk V, Wingert J, Dao Thi VL, Hu J, Schlegel P, Dieterich C, Katus HA, Frey N, Jungmann A, Ritterhoff J, Most P.   +25 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

Accelerated Adeno Associated Virus Upstream Process Development From High-Throughput Systems to Clinical Scale. [PDF]

Biotechnol Bioeng
Andaluz A, Monteverde B, Vera K, Tse B, Gajic I, Forelich C, Motevalian SP.   +6 more
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

Considerations for the Use of AAV‐based Gene Therapy in HIV‐Positive Individuals With Haemophilia

Haemophilia, EarlyView.
ABSTRACT Introduction There is a high prevalence of human immunodeficiency virus (HIV) infection among the haemophilia community due to treatment in the 1970s and 1980s with contaminated clotting factor. Lifelong treatment regimens for haemophilia and HIV are burdensome alone and pose a particular challenge for individuals living with both conditions ...
Jürgen K. Rockstroh, Julia C. Stingl, Marco Stadler, Anja Reichert, Heiner Wedemeyer, Johannes Oldenburg   +5 more
wiley   +1 more source

Functional orthogonality of parvoviral phospholipase A₂ domains in adeno-associated virus transduction. [PDF]

J Virol
Hull JA, Fusco RM, Tan J, Ochoa MA, Hall A, Wan X, Loeb E, Asokan A.   +7 more
europepmc   +1 more source