Results 21 to 30 of about 9,649,206 (293)

Progress in the use of adeno-associated viral vectors for gene therapy [PDF]

, 2004
The development of safe and efficient gene transfer vectors is crucial for the success of gene therapy trials. A viral vector system promising to meet these requirements is based on the apathogenic adeno-associated virus (AAV-2), a member of the ...
Markus Braun-Falco, Hallek, M., Michael Hallek, Buning, H., Braun-Falco, M., Hildegard Büning, Büning, H;Braun-Falco, M;Hallek, M, Büning, Hildegard;Braun-Falco, Markus;Hallek, Michael   +7 more
core   +1 more source

Capsid-specific removal of circulating antibodies to adeno-associated virus vectors. [PDF]

Sci Rep, 2020
International audienceNeutralizing antibodies directed against adeno-associated virus (AAV) are commonly found in humans. In seropositive subjects, vector administration is not feasible as antibodies neutralize AAV vectors even at low titers ...
Bertin B, Veron P, Leborgne C, Deschamps JY, Moullec S, Fromes Y, Collaud F, Boutin S, Latournerie V, van Wittenberghe L, Delache B, Le Grand R, Dereuddre-Bosquet N, Benveniste O, Moullier P, Masurier C, Merten O, Mingozzi F.   +17 more
europepmc   +2 more sources

Efficient delivery of Cre-recombinase to neurons in vivo and stable transduction of neurons using adeno-associated and lentiviral vectors [PDF]

, 2004
BACKGROUND: Inactivating genes in vivo is an important technique for establishing their function in the adult nervous system. Unfortunately, conventional knockout mice may suffer from several limitations including embryonic or perinatal lethality and the
Hermens, Wim T J M C, Lieberman, A., Eggers, R., Verhaagen, J., Sablitzky, Fred, Chakravarthy, Sridhara, Zhang, Jing Ying, Zhang, J.Y., Hermens, Wim TJMC, Zhang, Jing, Verhaagen, J, Ahmed, Bushra, Anderson, P.N., Eggers, R, Eggers, Ruben, Levelt, Christiaan, Chakravarthy, S, Levelt, C., Ahmed, BY, Verhaagen, Joost, Lieberman, A.R., Hermens, WTJMC, Lieberman, AR, Anderson, Patrick N., Ahmed, Bushra Y, Hermens, W.T., Niclou, SP, Anderson, PN, NICLOU, Simone P., Lieberman, A R, Zhang, JY, Anderson, Patrick N, Sablitzky, F., Levelt, C, Sablitzky, F, Niclou, S.P., Chakravarthy, S., Ahmed, B.Y.   +37 more
core   +1 more source

Potential for cellular stress response to hepatic factor VIII expression from AAV vector

Molecular Therapy: Methods & Clinical Development, 2016
Hemophilia A and B are coagulation disorders resulting from the loss of functional coagulation factor VIII (FVIII) or factor IX proteins, respectively.
Irene Zolotukhin, David M Markusic, Brett Palaschak, Brad E Hoffman, Meera A Srikanthan, Roland W Herzog   +5 more
doaj   +1 more source

Performance of AAV8 vectors expressing human factor IX from a hepatic-selective promoter following intravenous injection into rats [PDF]

, 2008
Background: Vectors based on adeno-associated virus-8 (AAV8) have shown efficiency and efficacy for liver-directed gene therapy protocols following intravascular injection, particularly in relation to haemophilia gene therapy. AAV8 has also been proposed
Amit Nathwani, Baker, AH, Lorraine M Work, Tracey Graham, McIntosh, JB, Jenny McIntosh, Nathwani, A, Work, LM, Graham, T, McIntosh, J, Andrew H Baker   +10 more
core   +1 more source

Gene Therapy Rescues Cardiac Dysfunction in Duchenne Muscular Dystrophy Mice by Elevating Cardiomyocyte Deoxy-Adenosine Triphosphate

JACC: Basic to Translational Science, 2019
Summary: Mutations in the gene encoding for dystrophin leads to structural and functional deterioration of cardiomyocytes and is a hallmark of cardiomyopathy in Duchenne muscular dystrophy (DMD) patients.
Stephen C. Kolwicz, Jr., PhD, John K. Hall, PhD, Farid Moussavi-Harami, MD, Xiolan Chen, PhD, Stephen D. Hauschka, PhD, Jeffrey S. Chamberlain, PhD, Michael Regnier, PhD, Guy L. Odom, PhD   +7 more
doaj   +1 more source

Subretinal delivery of adeno-associated virus serotype 2 results in minimal immune responses that allow repeat vector administration in immunocompetent mice [PDF]

, 2009
Background: Adeno-associated virus serotype 2 (AAV2) vectors show considerable promise for ocular gene transfer. However, one potential barrier to efficacious long-term therapy is the development of immune responses against the vector or transgene ...
Ali, R.R., Barker, S.E., Robbie, S.J., Smith, A.J., Natkunarajah, M., Buch, P., Balaggan, K.S., Duran, Y., MacLaren, R.E., Bainbridge, J.W. B., Broderick, C.A.   +10 more
core   +1 more source

Adeno-associated Virus as a Mammalian DNA Vector [PDF]

Microbiology Spectrum, 2015
ABSTRACT In the nearly five decades since its accidental discovery, adeno-associated virus (AAV) has emerged as a highly versatile vector system for both research and clinical applications. A broad range of natural serotypes, as well as an increasing number of capsid variants, has combined to produce a repertoire of vectors with different ...
Max, Salganik, Matthew L, Hirsch, Richard Jude, Samulski   +2 more
openaire   +2 more sources

Innate Immune Response to Viral Vectors in Gene Therapy

Viruses, 2023
Viral vectors play a pivotal role in the field of gene therapy, with several related drugs having already gained clinical approval from the EMA and FDA.
Yixuan Wang, Wenwei Shao
doaj   +1 more source

Perinatal Gene Transfer to the Liver [PDF]

, 2011
The liver acts as a host to many functions hence raising the possibility that any one may be compromised by a single gene defect. Inherited or de novo mutations in these genes may result in relatively mild diseases or be so devastating that death within
Buckley, SM, Ward, N.J., Buckley, S.M.K., Ward, NJ, Chan, J.K.Y., Waddington, S.N., Rahim, A.A., Waddington, SN, Rahim, AA, Chan, JK, Mckay, T.R., Howe, S.J., Howe, SJ, McKay, TR, Natalie J. Ward, Ahad A. Rahim, Steven J. Howe, Jerry K.Y.Chan, Suzanne M.K. Buckley, Simon N. Waddington, Tristan R. McKay   +20 more
core   +1 more source