Results 251 to 260 of about 214,719 (303)

Italian Patients Journey for Gene Therapy in Haemophilia A

Haemophilia, EarlyView.
ABSTRACT Introduction Gene therapy (GT) provides sustained FVIII levels without repeated infusions in Hemophilia A (HA) patients thus overcoming a major limitation of replacement therapy. However, issues remain e.g., patient selection criteria, duration and variability of transgene expression, quality of life and long‐term safety.
Giovanni Di Minno, Gaia Spadarella, Alessandra Borchiellini, Anna Borrelli, Andrea Buzzi, Giancarlo Castaman, Michele Cecchi, Raimondo De Cristofaro, Vincenzo La Mura, Angelo Lupi, Giuseppe Malcangi, Maria Elisa Mancuso, Maria Francesca Mansueto, Cristina Santoro, Rita Carlotta Santoro, Luigi Piero Solimeno, Annarita Tagliaferri, Ezio Zanon, Isabella Garagiola, Flora Peyvandi   +19 more
wiley   +1 more source

CapBuild: a cloud-native tool for adeno-associated virus capsid engineering. [PDF]

Nucleic Acids Res
Klein AH, Kuiper MJ, Burgess M, Wickramarachchi A, Jain Y, Bauer DC, Wilson L.   +6 more
europepmc   +1 more source

Unveiling Small Non-Coding RNA Dynamics During Recombinant Adeno-Associated Virus Production. [PDF]

Biotechnol J
Burkhart M, Langenbach K, Holzmann K, Hornung N, Baiz JA, Otte K.   +5 more
europepmc   +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

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

A rapid, visual, ultrasensitive and highly specific method for detecting adeno-associated virus 2020 based on the LAMP-CRISPR/Cas12a system. [PDF]

Poult Sci
Gong P, Tao D, Chen Q, Yang Y, Xie S, Chen X, Shi S, Wang Y, Wang L, Qian Y, Ye S.   +10 more
europepmc   +1 more source

The interaction between virus-bound KLF4 and host-bound PARP1 directs the localization of wild-type adeno-associated virus type 2 (wtAAV2) to cellular sites of DNA damage. [PDF]

J Virol
Abrahams RR, Larsen CIS, Ha R, Majumder K.   +3 more
europepmc   +1 more source

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Adeno-associated virus vectors

Current Opinion in Biotechnology, 1992
Adeno-associated virus is a human parvovirus that integrates its DNA genome into host cell chromosomes with very high efficiency. This suggests that adeno-associated virus may be a useful vector for human gene therapy. Interest in adeno-associated virus vectors increased greatly in the last year following reports that adeno-associated virus genome ...
Barrie J Carter
exaly   +3 more sources

Adeno-Associated Virus Biology

, 2011
Adeno-associated virus (AAV) was first discovered as a contaminant of adenovirus stocks in the 1960s. The development of recombinant AAV vectors (rAAV) was facilitated by early studies that generated infectious molecular clones, determined the sequence of the genome, and defined the genetic elements of the virus. The refinement of methods and protocols
Weitzman, Matthew D, Linden, R Michael
openaire   +4 more sources

Multimodal Production of Adeno-Associated Virus

, 2019
Adeno-associated virus (AAV) is an increasingly popular tool in the research laboratory, and use of this viral vector clinically is occurring at an accelerated pace. Nevertheless, despite its popularity, AAV is a relatively cumbersome virus to produce; however, significant efforts have been invested to develop, optimize, and simplify methodology that ...
Ivette M, Sandoval, Nathan M, Kuhn, Fredric P, Manfredsson   +2 more
openaire   +3 more sources