scholarly journals Vectors for Highly Efficient and Neuron-Specific Retrograde Gene Transfer for Gene Therapy of Neurological Diseases

10.5772/52611 ◽  
2013 ◽  
Author(s):  
Shigeki Kato ◽  
Kenta Kobayashi ◽  
Ken-ichi Inoue ◽  
Masahiko Takada ◽  
Kazuto Kobayashi
Keyword(s):  
Gene Therapy ◽  
Gene Transfer ◽  
Neurological Diseases ◽  
Highly Efficient ◽  
Retrograde Gene Transfer

scholarly journals Highly Efficient Retrograde Gene Transfer for Genetic Treatment of Neurological Diseases

10.5772/21992 ◽  
2011 ◽  
Author(s):  
Shigeki Kato ◽  
Masahito ◽  
Kenta Kobayashi ◽  
Ken-Ichi Inoue ◽  
Masahiko Takada ◽  
...  
Keyword(s):  
Gene Transfer ◽  
Neurological Diseases ◽  
Highly Efficient ◽  
Retrograde Gene Transfer

A Lentiviral Strategy for Highly Efficient Retrograde Gene Transfer by Pseudotyping with Fusion Envelope Glycoprotein

2011 ◽  
Vol 22 (2) ◽  
pp. 197-206 ◽  
Author(s):  
Shigeki Kato ◽  
Kenta Kobayashi ◽  
Ken-ichi Inoue ◽  
Masahito Kuramochi ◽  
Tomoaki Okada ◽  
...  
Keyword(s):  
Gene Transfer ◽  
Envelope Glycoprotein ◽  
Highly Efficient ◽  
Retrograde Gene Transfer

scholarly journals Highly Efficient Retrograde Gene Transfer into Motor Neurons by a Lentiviral Vector Pseudotyped with Fusion Glycoprotein

PLoS ONE ◽  
2013 ◽  
Vol 8 (9) ◽  
pp. e75896 ◽  
Author(s):  
Miyabi Hirano ◽  
Shigeki Kato ◽  
Kenta Kobayashi ◽  
Tomoaki Okada ◽  
Hiroyuki Yaginuma ◽  
...  
Keyword(s):  
Gene Transfer ◽  
Motor Neurons ◽  
Lentiviral Vector ◽  
Highly Efficient ◽  
Fusion Glycoprotein ◽  
Retrograde Gene Transfer

Application of highly efficient retrograde gene transfer vector for primate brain research

2011 ◽  
Vol 71 ◽  
pp. e41
Author(s):  
Kazuto Kobayashi ◽  
Shigeki Kato ◽  
Masahito Kuramochi ◽  
Kenta Kobayashi ◽  
Ken-ichi Inoue ◽  
...  
Keyword(s):  
Gene Transfer ◽  
Brain Research ◽  
Transfer Vector ◽  
Highly Efficient ◽  
Primate Brain ◽  
Gene Transfer Vector ◽  
Retrograde Gene Transfer

Highly Efficient mRNA- and cDNA-Based Transient Gene Delivery into Human Progenitor Cells.

Blood ◽  
2006 ◽  
Vol 108 (11) ◽  
pp. 5471-5471
Author(s):  
Jochen Greiner ◽  
Jan Torzewski ◽  
Peter Ponsaerts ◽  
Markus T. Rojewski ◽  
Desiree Kronawitter ◽  
...  
Keyword(s):  
Tissue Engineering ◽  
Gene Therapy ◽  
Stem Cell ◽  
Gene Transfer ◽  
Progenitor Cells ◽  
Transfection Efficiency ◽  
Highly Efficient ◽  
Human Cd34

Abstract The method of gene transfer into progenitor cells is critical as viral vector transduction involves the risk of tumor induction by non-specific genomic integration. Non-viral transfection systems often fail due to low transfection efficiency. However, gene transfer into human CD34+ hematopoietic progenitor (HPC) and mesenchymal stem cells (MSC) is an essential tool for in vitro- and in vivo-applications and therapeutic strategies such as tissue engineering and gene therapy. We recently reported an transient genetic labelling of human CD34+ HPC with deltaLNGFR-plasmid-DNA for in vivo application: Transient transfection was efficient for both, CD34+ HSC (41% ± 2%) and leukemia cell lines (55% ± 4.9%) using the method of nucleofection. Moreover, mature myeloid cells (CD66b+) derived from transfected human CD34+ HPC and leukemia cells maintained deltaLNGFR expression at a high percentage (70% ± 1.6% and 58% ± 2% respectively). In this work, we investigated labelling of CD34+ HPC with mRNA. Human CD34+ HPC and human MSC were transfected with in vitro-transcribed mRNA for deltaLNGFR, a marker gene approved for human in vivo-application, using nucleofection. EGFP was used as a control. 24h after nucleofection, FACS-analysis showed a higher transfection efficiency compared to plasmid transfected CD34+ HPC and MSC: A high transfection frequency was found for mRNA-transfected HPCs using deltaLNGFR (82.4±9.7%) and EGFP (88.7±2.6%). We found also a high transfection rate for MSC using the marker genes deltaLNGFR (92.4±3.6%) and EGFP (83.3±4.1%). Cell viability was not affected by mRNA-transfection. Moreover, differentiation assays of deltaLNGFR-selected MSC after transfection, showed that differentiation of MSC into mesenchymal cells like chondrocytes, adipocytes and osteoblasts was not affected by mRNA nucleofection. Taken together, mRNA based nucleofection is a powerful, highly efficient and non-toxic approach for transient labelling of human progenitor cells or, via transfection of selective proteins, for transient manipulation of stem cell function. It may thus be useful to transiently manipulate stem cell characteristics and combine principles of gene therapy and tissue engineering.

Therapeutic Applications of Human Heme Oxygenase Gene Transfer and Gene Therapy

2003 ◽  
Vol 9 (30) ◽  
pp. 2513-2524 ◽  
Author(s):  
Nader Abraham
Keyword(s):  
Gene Therapy ◽  
Gene Transfer ◽  
Heme Oxygenase ◽  
Therapeutic Applications

Gene Therapy in Fanconi Anemia: A Matter of Time, Safety and Gene Transfer Tool Efficiency

2017 ◽  
Vol 16 (5) ◽  
pp. 297-308 ◽  
Author(s):  
Els Verhoeyen ◽  
Francisco Roman-Rodriguez ◽  
Francois-Loic Cosset ◽  
Camille Levy ◽  
Paula Rio
Keyword(s):  
Gene Therapy ◽  
Gene Transfer ◽  
Fanconi Anemia

scholarly journals Achieving highly efficient gene transfer to the bladder by increasing the molecular weight of polymer-based nanoparticles

2021 ◽  
Vol 332 ◽  
pp. 210-224
Author(s):  
Gang Li ◽  
Shanshan He ◽  
Andreas G. Schätzlein ◽  
Robert M. Weiss ◽  
Darryl T. Martin ◽  
...  
Keyword(s):  
Molecular Weight ◽  
Gene Transfer ◽  
Highly Efficient ◽  
Efficient Gene

scholarly journals Retrograde Transgene Expression via Neuron-Specific Lentiviral Vector Depends on Both Species and Input Projections

Viruses ◽  
2021 ◽  
Vol 13 (7) ◽  
pp. 1387
Author(s):  
Yukiko Otsuka ◽  
Hitomi Tsuge ◽  
Shiori Uezono ◽  
Soshi Tanabe ◽  
Maki Fujiwara ◽  
...  
Keyword(s):  
Gene Transfer ◽  
Immune Responses ◽  
Envelope Glycoprotein ◽  
Transgene Expression ◽  
Histological Analysis ◽  
Lentiviral Vector ◽  
Cortical Input ◽  
Input System ◽  
Vesicular Stomatitis ◽  
Retrograde Gene Transfer

For achieving retrograde gene transfer, we have so far developed two types of lentiviral vectors pseudotyped with fusion envelope glycoprotein, termed HiRet vector and NeuRet vector, consisting of distinct combinations of rabies virus and vesicular stomatitis virus glycoproteins. In the present study, we compared the patterns of retrograde transgene expression for the HiRet vs. NeuRet vectors by testing the cortical input system. These vectors were injected into the motor cortex in rats, marmosets, and macaques, and the distributions of retrograde labels were investigated in the cortex and thalamus. Our histological analysis revealed that the NeuRet vector generally exhibits a higher efficiency of retrograde gene transfer than the HiRet vector, though its capacity of retrograde transgene expression in the macaque brain is unexpectedly low, especially in terms of the intracortical connections, as compared to the rat and marmoset brains. It was also demonstrated that the NeuRet but not the HiRet vector displays sufficiently high neuron specificity and causes no marked inflammatory/immune responses at the vector injection sites in the primate (marmoset and macaque) brains. The present results indicate that the retrograde transgene efficiency of the NeuRet vector varies depending not only on the species but also on the input projections.

Highly efficient cell-mediated gene transfer using non-viral vectors and FuGene™6: in vitro and in vivo studies

2000 ◽  
Vol 57 (8) ◽  
pp. 1326-1333 ◽  
Author(s):  
I. Hellgren* ◽  
V. Drvota ◽  
R. Pieper ◽  
S. Enoksson ◽  
P. Blomberg ◽  
...  
Keyword(s):  
Gene Transfer ◽  
Viral Vectors ◽  
In Vivo Studies ◽  
Highly Efficient
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