Tailoring highly branched poly(β-amino ester)s: a synthetic platform for epidermal gene therapy

2015 ◽  
Vol 51 (40) ◽  
pp. 8473-8476 ◽  
Author(s):  
Jian-Yuan Huang ◽  
Yongsheng Gao ◽  
Lara Cutlar ◽  
Jonathan O'Keeffe-Ahern ◽  
Tianyu Zhao ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Gene Delivery ◽  
Human Keratinocytes ◽  
Amino Ester ◽  
Efficient Gene

Highly branched poly(β-amino ester)s were synthesised for safer and more efficient gene delivery to human keratinocytes than commercial transfection reagents.

Advanced Materials for Gene Delivery

2014 ◽  
Vol 995 ◽  
pp. 29-47 ◽  
Author(s):  
Mohammad A. Jafar Mazumder ◽  
Md. Hasan Zahir ◽  
Sharif F. Zaman
Keyword(s):  
Gene Therapy ◽  
Gene Delivery ◽  
Genetic Disorders ◽  
Basic Research ◽  
Advanced Materials ◽  
Clinical Settings ◽  
Efficient Gene ◽  
Efficient Delivery ◽  
Promising Treatment

Gene therapy is a widespread and promising treatment of many diseases resulting from genetic disorders, infections and cancer. The feasibility of the gene therapy is mainly depends on the development of appropriate method and suitable vectors. For an efficient gene delivery, it is very important to use a carrier that is easy to produce, stable, non-oncogenic and non-immunogenic. Currently most of the vectors actually suffer from many problems. Therefore, the ideal gene therapy delivery system should be developed that can be easily used for highly efficient delivery and able to maintain long-term gene expression, and can be applicable to basic research as well as clinical settings. This article provides a brief over view on the concept and aim of gene delivery, the different gene delivery systems and use of different materials as a carrier in the area of gene therapy.

Multivalent polyrotaxane vectors as adaptive cargo complexes for gene therapy

2018 ◽  
Vol 9 (7) ◽  
pp. 845-859 ◽  
Author(s):  
Rodinel Ardeleanu ◽  
Andrei I. Dascalu ◽  
Andrei Neamtu ◽  
Dragos Peptanariu ◽  
Cristina M. Uritu ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Gene Delivery ◽  
Delivery Systems ◽  
Efficient Gene

The philosophy to design and construct polyrotaxane carriers, as efficient gene delivery systems.

Biocompatible fluorinated poly(β-amino ester)s for safe and efficient gene therapy

2018 ◽  
Vol 535 (1-2) ◽  
pp. 180-193 ◽  
Author(s):  
Jia-Hui Gong ◽  
Yi Wang ◽  
Lei Xing ◽  
Peng-Fei Cui ◽  
Jian-Bin Qiao ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Amino Ester ◽  
Efficient Gene

Ligand targeting and peptide functionalized polymers as non-viral carriers for gene therapy

2020 ◽  
Vol 8 (1) ◽  
pp. 64-83 ◽  
Author(s):  
Khan Muhammad ◽  
Jing Zhao ◽  
Ihsan Ullah ◽  
Jintang Guo ◽  
Xiang-kui Ren ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Gene Delivery ◽  
Functionalized Polymers ◽  
Gene Carriers ◽  
Efficient Gene

Ligand targeting and peptide functionalized polymers serve as gene carriers for efficient gene delivery.

Selective and Efficient Gene Delivery of CD40-Ligand by a Fiber-Modified Adenovirus Vector with Specific Antibody to Human Leukemia and Myeloma.

Blood ◽  
2004 ◽  
Vol 104 (11) ◽  
pp. 5263-5263
Author(s):  
Kazunori Kato ◽  
Sachie Hirai ◽  
Yukari Masuta ◽  
Aiko Kuroishi ◽  
Kiminori Nakamura ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Immune Response ◽  
B Cells ◽  
Gene Delivery ◽  
Tumor Cells ◽  
Specific Antibody ◽  
Adenovirus Vector ◽  
Cd40 Ligand ◽  
Myeloma Cells ◽  
Efficient Gene

Abstract The use of adenovirus vector for cancer gene therapy is limited by their low transduction efficiency for lymphoma, leukemia and myeloma. We previously reported that highly efficient gene delivery of CD40-ligand by a modified adenovirus vector with the integrin-binding motif, RGD, in the H1 loop of the fiber knob (AxCAhCD40L-F/RGD) could induce phenotypic alteration followed by T cell immune response to autologous leukemia cells. But the utility of adenovirus with RGD-motif is still limited by their lack of specificity on tumor cells. Recent studies revealed a novel strategy of targeting adenovirus using a bispecific single-chain antibody (scFv) specific for adenovirus and target molecules on tumor cell surface. However, this approach should permit the production of high quantities of active bispecific scFv for in vivo use. To target adenovirus to hematopoietic tumor cells efficiently, we herein constructed a modified adenovirus vector that contained a synthetic immunoglobulin G-binding domain (termed Z33) in H1 loop of the fiber knob. A recombinant adenovirus encoding EGFP, lacZ (as reporter gene; Ax3CAZ3-F/Z33 or Ax3EGFP-F/Z33) or CD40L (as a therapeutic gene; Ax3CD40L-F/Z33) with Z33-modified fiber were tested for gene transfer efficiency into human tumors such as lymphoma, leukemia and myeloma cells. By the treatment with various antibodies specific for CD20 (Rituximab), CD40, CD38, CCR2 or CXCR4 that are expressed on leukemic cells, we achieved 3 to 10-fold enhancement of gene expression in lymphoma/leukemia (Ramos, Daudi or THP-1) and myeloma cells (MM1S, IM-9 or KMS5) compared with control IgG-treated tumors. We also examined specific gene delivery to freshly isolated leukemia B cells from patients that also contains normal lymphocytes. By using antibody to CD20 or CD40, we could selectively deliver CD40L gene with Ax3CD40L-F/Z33 into leukemia B cells (>50% at 300 pu/cell), but not in T and monocytes, followed by the induction of immune costimulatory molecules that are important in anti-leukemia immune response. Overall, our results indicated that combination of Z33-modified adenovirus vector and tumor specific antibody can be used as a modality for the gene therapy of leukemia and myeloma.

scholarly journals Glycosylated Artificial Virus-Like Hybrid Vectors for Advanced Gene Delivery

Polymers ◽  
2019 ◽  
Vol 11 (2) ◽  
pp. 243 ◽  
Author(s):  
Shashank Pinnapireddy ◽  
Mohamed Raafat El Assy ◽  
Patrick Schlote ◽  
Udo Bakowsky
Keyword(s):  
Gene Therapy ◽  
Gene Delivery ◽  
Transfection Efficiency ◽  
Viral Envelope ◽  
Specific Gene ◽  
Cationic Polymers ◽  
Hybrid Vectors ◽  
Efficient Gene ◽  
Delivery Vehicles ◽  
Viral Envelopes

The major obstacle facing efficient gene therapy is the development of reliable delivery vehicles, which are both nontoxic and biocompatible and possess efficient cell-specific gene delivery. Previously, hybrid delivery vehicles comprising anionic liposomes and cationic polymers have been used successfully for gene therapy. In this study, hybrid vectors based on glycosylated artificial viral envelopes (including two novel compositions mimicking HIV and HSV envelopes) and polyethylenimine were morphologically and physiologically characterised. Transfection studies showed that the hybrid vectors based on the control liposomes, and their glycosylated modifications, had significantly higher transfection rates compared to the polyplexes. Improvement in the transfection efficiency was observed with the glycosylated HIV- and HSV-mimicking hybrid vectors, which also showed a safe biocompatibility profile based on the cytotoxicity and haemocompatibility assays. These glycosylated artificial viral envelope-based hybrid vectors could be used as safe gene delivery systems with potential to become new compositions for efficient nonviral gene therapy.

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