[PDF][PDF] Autologous, gene-modified hematopoietic stem and progenitor cells repopulate the central nervous system with distinct clonal variants

CW Peterson, JE Adair, ME Wohlfahrt, C Deleage… - Stem cell reports, 2019 - cell.com
to target gene-modified cells to tissues. Gene-marked … and fluorescent bead uptake in
CD45+Lin–HLA-DR+ cells. We refer … -fluorescent staining with antibodies against GFP (green) and …

Hematopoietic gene therapies for metabolic and neurologic diseases

A Biffi - Hematology/Oncology Clinics, 2017 - hemonc.theclinics.com
… metabolic diseases affecting the central nervous system (CNS)… (ALS), receive hematopoietic
cell transplantation (HCT) from … The rationale for the use of HCT to treat these disorders is …

[HTML][HTML] … nanoparticles and an exogenous gene into the acutely inflamed brain tissue–evaluations on monocyte-based delivery system for the central nervous system

HI Tong, W Kang, PMC Davy, Y Shi, S Sun… - PLoS …, 2016 - journals.plos.org
… Early studies using hematopoietic stem cell (HSC) … utilized to mediate the transfer of green
fluorescent protein (GFP) gene … more suitable to be used as cell vehicles targeting the brain …

[HTML][HTML] Hematopoietic stem cell gene therapy ameliorates CNS involvement in murine model of GM1-gangliosidosis

T Tsunogai, T Ohashi, Y Shimada, T Higuchi… - … Therapy-Methods & …, 2022 - cell.com
… ex vivo gene therapy targeting hematopoietic stem cells using a … activity in the central nervous
system and visceral organs. … EGFP fluorescence was observed in the cerebral cortex and …

Genetic Modification of Mesenchymal Stem Cells for Neurological Disease Therapy: What Effects Does it Have on Phenotype/Cell Behavior, Determining Their …

N Ebrahim, V James, AA Rizvanov… - Molecular Diagnosis & …, 2020 - Springer
… the central nervous system by improving neurological function, … However, given the diversity
of stem cells and disease targetsGreen fluorescent protein is widely used as a reporter gene …

[HTML][HTML] … marrow-derived macrophages and the CNS: An update on the use of experimental chimeric mouse models and bone marrow transplantation in neurological …

A Larochelle, MA Bellavance, JP Michaud… - Biochimica et Biophysica …, 2016 - Elsevier
… The central nervous system (CNS) is a very unique system … For an optimal reconstitution
of the hematopoietic system by … T cells were shown to target transplanted hematopoietic

Regulatable lentiviral hematopoietic stem cell gene therapy in a mouse model of Parkinson's disease

G Ge, C Chen, MJ Guderyon, J Liu, Z He… - Stem cells and …, 2018 - liebertpub.com
… circulating monocytes into the central nervous system (CNS). … The images were captured
with a fluorescent microscope (… to express and deliver GDNF to target neurons effectively. We …

[HTML][HTML] Gene-modified leucoconcentrate for personalized ex vivo gene therapy in a mini pig model of moderate spinal cord injury

RR Islamov, FV Bashirov, ME Sokolov… - Neural Regeneration …, 2021 - journals.lww.com
… and fluorescent microscopy studies of the gene-modified … The method was improved to use
hematopoietic stem cells (… of using leucocytes for targeted delivery of recombinant genes that …

Targeted gene addition at the CX3CR1 locus of human HSPCs improves their ability to repopulate the myeloid CNS compartment and drives lineage specific and …

A Biffi, A Montepeloso, D Mattioli, D Pellin, M Peviani… - 2023 - researchsquare.com
… Cx3cr1-/+ versus WT cells within hematopoietic cell lineages … replace endogenous microglia
with gene modified MLCs upon … sections, we used a fluorescence microscope Delta Vision …

[HTML][HTML] Improved engraftment and therapeutic efficacy by human genome-edited hematopoietic stem cells with Busulfan-based myeloablation

E Poletto, P Colella, LNP Vera, S Khan… - … Therapy-Methods & …, 2022 - cell.com
… Among these tools, CRISPR-Cas9 has been widely adopted as it uses an RNA molecule
(gRNA) to target the nuclease (Cas9) to the desired genomic locus. Cas9 binding and the …