Results 21 to 30 of about 145,799 (355)

Rescue of splicing-mediated intron loss maximizes expression in lentiviral vectors containing the human ubiquitin C promoter. [PDF]

, 2014
Lentiviral vectors almost universally use heterologous internal promoters to express transgenes. One of the most commonly used promoter fragments is a 1.2-kb sequence from the human ubiquitin C (UBC) gene, encompassing the promoter, some enhancers, first
Cooper, Aaron R, Gschweng, Eric H, Kohn, Donald B, Lill, Georgia R   +3 more
core   +1 more source

Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway. [PDF]

, 2003
A systematic evaluation of the influence of sex on transduction by recombinant adeno-associated viral vector (rAAV) indicated that transgene expression after liver-targeted delivery of vector particles was between 5- to 13-fold higher in male mice ...
Davidoff, AM, Nathwani, AC, Ng, CYC, Spence, Y, Zhou, J   +4 more
core   +1 more source

Efficient lentiviral transduction method to gene modify cord blood CD8+ T cells for cancer therapy applications

Molecular Therapy: Methods & Clinical Development, 2021
Adoptive T cell therapy utilizing tumor-specific autologous T cells has shown promising results for cancer treatment. However, the limited numbers of autologous tumor-associated antigen (TAA)-specific T cells and the functional aberrancies, due to ...
Vania Lo Presti, Annelisa M. Cornel, Maud Plantinga, Ester Dünnebach, Jurgen Kuball, Jaap Jan Boelens, Stefan Nierkens, Niek P. van Til   +7 more
doaj   +1 more source

Factors influencing in vivo transduction by recombinant adeno-associated viral vectors expressing the human factor IX cDNA. [PDF]

, 2001
Long-term expression of coagulation factor IX (FIX) has been observed in murine and canine models following administration of recombinant adeno-associated viral (rAAV) vectors into either the portal vein or muscle. These studies were designed to evaluate
Davidoff, A, Hanawa, H, Nathwani, AC, Nienhuis, AW, Vanin, EF, Zhou, JF   +5 more
core   +1 more source

Enhancing transduction of the liver by adeno-associated viral vectors [PDF]

Gene Therapy, 2008
A number of distinct factors acting at different stages of the adeno-associated virus vector (AAV)-mediated gene transfer process were found to influence murine hepatocyte transduction. Foremost among these was the viral capsid protein. Self-complementary (sc) AAV pseudotyped with capsid from serotype 8 or rh.10 mediated fourfold greater hepatocyte ...
Nathwani, A. C., Cochrane, M., McIntosh, J., Ng, C. Y. C., Zhou, J., Gray, J. T., Davidoff, A. M.   +6 more
openaire   +3 more sources

Tetrameric architecture of an active phenol-bound form of the AAA+ transcriptional regulator DmpR

Nature Communications, 2020
DmpR is a bacterial enhancer binding protein from the AAA+ family of ATPases that binds aromatic compounds and controls the transcription of genes involved in the degradation of toxic pollutants.
Kwang-Hyun Park, Sungchul Kim, Su-Jin Lee, Jee-Eun Cho, Vinod Vikas Patil, Arti Baban Dumbrepatil, Hyung-Nam Song, Woo-Chan Ahn, Chirlmin Joo, Seung-Goo Lee, Victoria Shingler, Eui-Jeon Woo   +11 more
doaj   +1 more source

Infectivity enhancement for adenoviral transduction of canine osteosarcoma cells [PDF]

Gene Therapy, 2005
The full realization of conditionally replicative adenoviruses (CRAds) for cancer therapy has been hampered by the limited knowledge of CRAd function in vivo and particularly in an immunocompetent host. To address this issue, we previously proposed a canine adenovirus type 2 (CAV2)-based CRAd for clinical evaluation in canine patients with osteosarcoma
L P, Le, A A, Rivera, J N, Glasgow, V V, Ternovoi, H, Wu, M, Wang, B F, Smith, G P, Siegal, D T, Curiel   +8 more
openaire   +2 more sources

The Optimized γ-Globin Lentiviral Vector GGHI-mB-3D Leads to Nearly Therapeutic HbF Levels In Vitro in CD34⁺ Cells from Sickle Cell Disease Patients

Viruses, 2022
We have previously demonstrated that both the original γ-globin lentiviral vector (LV) GGHI and the optimized GGHI-mB-3D LV, carrying the novel regulatory elements of the 3D HPFH-1 enhancer and the 3’ β-globin UTR, can significantly increase HbF ...
Ekati Drakopoulou, Maria Georgomanoli, Carsten W. Lederer, Fottes Panetsos, Marina Kleanthous, Ersi Voskaridou, Dimitrios Valakos, Eleni Papanikolaou, Nicholas P. Anagnou   +8 more
doaj   +1 more source

Role of cyclic AMP in the control of cell-specific gene expression [PDF]

, 1993
Genes have to be expressed in specific cell types at appropriate times of development dependent on external signals. cAMP signaling occurs in all cells, thus raising the question of how this signal transduction pattern is integrated into mechanisms ...
Boshart, Michael, Nitsch, Doris, Schmid, Wolfgang, Schütz, Günther   +3 more
core   +1 more source

Translating the combination of gene therapy and tissue engineering for treating recessive dystrophic epidermolysis bullosa [PDF]

European Cells & Materials, 2018
The combination of gene therapy and tissue engineering is one of the most promising strategies for the treatment of recessive dystrophic epidermolysis bullosa (RDEB).
A Dakiw Piaceski, D Larouche, K Ghani, F Bisson, S Cortez Ghio, S Larochelle, VJ Moulin, M Caruso, L Germain   +8 more
doaj   +1 more source