In vivo HSC prime editing rescues sickle cell disease in a mouse model
Sickle cell disease (SCD) is a monogenic disease caused by a nucleotide mutation in the β-
globin gene. Current gene therapy studies are mainly focused on lentiviral vector–mediated …
globin gene. Current gene therapy studies are mainly focused on lentiviral vector–mediated …
Repair mechanisms help glioblastoma resist treatment
Glioblastoma multiforme (GBM) is a malignant and incurable glial brain tumour. The current
best treatment for GBM includes maximal safe surgical resection followed by concomitant …
best treatment for GBM includes maximal safe surgical resection followed by concomitant …
Genetic strategies for HIV treatment and prevention
A Falkenhagen, S Joshi - Molecular Therapy-Nucleic Acids, 2018 - cell.com
Conventional HIV gene therapy approaches are based on engineering HIV target cells that
are non-permissive to viral replication. However, expansion of gene-modified HIV target …
are non-permissive to viral replication. However, expansion of gene-modified HIV target …
Development of hematopoietic stem cell based gene therapy for HIV-1 infection: considerations for proof of concept studies and translation to standard medical …
Over the past 15 years we have been investigating an alternative approach to treating HIV-
1/AIDS, based on the creation of a disease-resistant immune system through transplantation …
1/AIDS, based on the creation of a disease-resistant immune system through transplantation …
A combined in vivo HSC transduction/selection approach results in efficient and stable gene expression in peripheral blood cells in mice
We recently reported on an in vivo hematopoietic stem cell (HSC) gene therapy approach. It
involves the subcutaneous injections of G-CSF/AMD3100 to mobilize HSCs from the bone …
involves the subcutaneous injections of G-CSF/AMD3100 to mobilize HSCs from the bone …
Stable HIV decoy receptor expression after in vivo HSC transduction in mice and NHPs: Safety and efficacy in protection from SHIV
C Li, AK Anderson, H Wang, S Gil, J Kim, L Huang… - Molecular Therapy, 2023 - cell.com
We aim to develop an in vivo hematopoietic stem cell (HSC) gene therapy approach for
persistent control/protection of HIV-1 infection based on the stable expression of a secreted …
persistent control/protection of HIV-1 infection based on the stable expression of a secreted …
Optimized lentiviral vectors for HIV gene therapy: multiplexed expression of small RNAs and inclusion of MGMTP140K drug resistance gene
J Chung, LJ Scherer, A Gu, AM Gardner… - Molecular Therapy, 2014 - cell.com
Gene therapy with hematopoietic stem and progenitor cells is a promising approach to
engineering immunity to human immunodeficiency virus (HIV) that may lead to a functional …
engineering immunity to human immunodeficiency virus (HIV) that may lead to a functional …
In Vivo Gene Therapy for Canine SCID-X1 Using Cocal-Pseudotyped Lentiviral Vector
Hematopoietic stem and progenitor cell (HSPC)-based ex vivo gene therapy has
demonstrated clinical success for X-linked severe combined immunodeficiency (SCID-X1) …
demonstrated clinical success for X-linked severe combined immunodeficiency (SCID-X1) …
211Astatine-Conjugated Monoclonal CD45 Antibody-Based Nonmyeloablative Conditioning for Stem Cell Gene Therapy
CR Burtner, D Chandrasekaran, EB Santos… - Human Gene …, 2015 - liebertpub.com
Most hematopoietic stem cell gene therapy studies require host conditioning to allow for
efficient engraftment of gene-modified cells. Conditioning regimens with lower treatment …
efficient engraftment of gene-modified cells. Conditioning regimens with lower treatment …
Assessment of biomarkers influencing treatment success on small intestinal lymphoma in dogs
H Yamazaki, H Sasai, M Tanaka… - Veterinary and …, 2021 - Wiley Online Library
This study aimed to determine a reliable therapeutic biomarker for localized small intestinal
lymphoma (SIL) in dogs based on clinical and histopathological features. We retrospectively …
lymphoma (SIL) in dogs based on clinical and histopathological features. We retrospectively …