Viral vector platforms within the gene therapy landscape

JT Bulcha, Y Wang, H Ma, PWL Tai… - Signal transduction and …, 2021 - nature.com
Throughout its 40-year history, the field of gene therapy has been marked by many
transitions. It has seen great strides in combating human disease, has given hope to patients …

Hiv dna integration

R Craigie, FD Bushman - Cold Spring …, 2012 - perspectivesinmedicine.cshlp.org
Retroviruses are distinguished from other viruses by two characteristic steps in the viral
replication cycle. The first is reverse transcription, which results in the production of a double …

Capsid-CPSF6 interaction licenses nuclear HIV-1 trafficking to sites of viral DNA integration

V Achuthan, JM Perreira, GA Sowd, M Puray-Chavez… - Cell Host & Microbe, 2018 - cell.com
HIV-1 integration into the host genome favors actively transcribed genes. Prior work
indicated that the nuclear periphery provides the architectural basis for integration site …

Structure and function of retroviral integrase

GN Maertens, AN Engelman… - Nature Reviews …, 2022 - nature.com
A hallmark of retroviral replication is establishment of the proviral state, wherein a DNA copy
of the viral RNA genome is stably incorporated into a host cell chromosome. Integrase is the …

Gene therapy targeting haematopoietic stem cells for inherited diseases: progress and challenges

M Cavazzana, FD Bushman, A Miccio… - Nature reviews Drug …, 2019 - nature.com
Pioneering gene therapy trials have shown that the genetic engineering of haematopoietic
stem and progenitor cells can be an alternative to allogeneic transplantation in the treatment …

A critical role for alternative polyadenylation factor CPSF6 in targeting HIV-1 integration to transcriptionally active chromatin

GA Sowd, E Serrao, H Wang, W Wang… - Proceedings of the …, 2016 - National Acad Sciences
Integration is vital to retroviral replication and influences the establishment of the latent HIV
reservoir. HIV-1 integration favors active genes, which is in part determined by the …

Retroviral DNA integration

P Lesbats, AN Engelman, P Cherepanov - Chemical reviews, 2016 - ACS Publications
The integration of a DNA copy of the viral RNA genome into host chromatin is the defining
step of retroviral replication. This enzymatic process is catalyzed by the virus-encoded …

Psip1/Ledgf p52 binds methylated histone H3K36 and splicing factors and contributes to the regulation of alternative splicing

MM Pradeepa, HG Sutherland, J Ule, GR Grimes… - PLoS …, 2012 - journals.plos.org
Increasing evidence suggests that chromatin modifications have important roles in
modulating constitutive or alternative splicing. Here we demonstrate that the PWWP domain …

Host cell factors in HIV replication: meta-analysis of genome-wide studies

FD Bushman, N Malani, J Fernandes, I D'Orso… - PLoS …, 2009 - journals.plos.org
We have analyzed host cell genes linked to HIV replication that were identified in nine
genome-wide studies, including three independent siRNA screens. Overlaps among the …

CD19-targeting CAR T cell immunotherapy outcomes correlate with genomic modification by vector integration

CL Nobles, S Sherrill-Mix, JK Everett… - The Journal of …, 2020 - Am Soc Clin Investig
Chimeric antigen receptor–engineered T cells targeting CD19 (CART19) provide an
effective treatment for pediatric acute lymphoblastic leukemia but are less effective for …