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Pharmaceutics
Special Issues
Scaffold-Mediated Gene Delivery
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Scaffold-Mediated Gene Delivery

A special issue of Pharmaceutics (ISSN 1999-4923). This special issue belongs to the section "Gene and Cell Therapy".

Deadline for manuscript submissions: closed (10 June 2020) | Viewed by 10273

Special Issue Editor

Dr. Ana Rey-Rico

E-Mail Website
Guest Editor
Centro de Investigacións Científicas Avanzadas (CICA), Universidade da Coruña, Campus de A Coruña, 15071 A Coruña, Spain
Interests: drug delivery; gene therapy; biomaterials; regenerative medicine

Special Issue Information

Dear Colleagues,

Gene therapy constitutes one of the most promising therapeutic strategies in the treatment of several chronic or genetic disorders. 

Current gene therapy approaches mainly focus on providing a safe gene supply and an effective transgene expression into a desired location. At the interface of gene therapy drug delivery and biomaterials science, gene delivery has emerged as a powerful tool to achieve this goal. Use of scaffolds as delivery systems permits obtaining a controlled, sustained, and localized release of the vectors encoding for therapeutic factors into a specific target. Likewise, other concerns associated with the direct administration of the gene transfer vectors as safety or immunogenicity (viral vectors) or low efficiency (non-viral vectors) can be avoided. 

The design of biomaterials acting as a vector reservoir of an efficient gene transfer and exhibiting adapted properties to the target tissue is therefore the focus of active investigation. In this regard, different scaffolds including hydrogels or solid matrices have been studied as gene delivery platforms for promoting the regeneration of complex tissues, such as cartilage, skin, blood vessels, and nerves. 

This Special Issue has the aim of highlighting current progress on the use of scaffolds as gene delivery systems in different tissue engineering and regenerative medicine approaches.  

Dr. Ana Rey-Rico
Guest Editor

Manuscript Submission Information

Manuscripts should be submitted online at www.mdpi.com by registering and logging in to this website. Once you are registered, click here to go to the submission form. Manuscripts can be submitted until the deadline. All submissions that pass pre-check are peer-reviewed. Accepted papers will be published continuously in the journal (as soon as accepted) and will be listed together on the special issue website. Research articles, review articles as well as short communications are invited. For planned papers, a title and short abstract (about 100 words) can be sent to the Editorial Office for announcement on this website.

Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). All manuscripts are thoroughly refereed through a single-blind peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. Pharmaceutics is an international peer-reviewed open access monthly journal published by MDPI.

Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 2900 CHF (Swiss Francs). Submitted papers should be well formatted and use good English. Authors may use MDPI's English editing service prior to publication or during author revisions.

Keywords

  • Gene therapy
  • Gene delivery
  • Viral vectors
  • Non-viral vectors
  • Scaffolds
  • Hydrogels
  • Gene activated matrices
  • Transgene expression
  • Tissue regeneration

Published Papers (3 papers)

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Research

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17 pages, 1307 KiB  
Article
Controlled Release of rAAV Vectors from APMA-Functionalized Contact Lenses for Corneal Gene Therapy
by Fernando Alvarez-Rivera, Ana Rey-Rico, Jagadeesh K Venkatesan, Luis Diaz-Gomez, Magali Cucchiarini, Angel Concheiro and Carmen Alvarez-Lorenzo
Pharmaceutics 2020, 12(4), 335; https://doi.org/10.3390/pharmaceutics12040335 - 9 Apr 2020
Cited by 17 | Viewed by 3573
Abstract
As an alternative to eye drops and ocular injections for gene therapy, the aim of this work was to design for the first time hydrogel contact lenses that can act as platforms for the controlled delivery of viral vectors (recombinant adeno-associated virus, rAAV) [...] Read more.
As an alternative to eye drops and ocular injections for gene therapy, the aim of this work was to design for the first time hydrogel contact lenses that can act as platforms for the controlled delivery of viral vectors (recombinant adeno-associated virus, rAAV) to the eye in an effective way with improved patient compliance. Hydrogels of hydroxyethyl methacrylate (HEMA) with aminopropyl methacrylamide (APMA) (H1: 40, and H2: 80 mM) or without (Hc: 0 mM) were synthesized, sterilized by steam heat (121 °C, 20 min), and then tested for gene therapy using rAAV vectors to deliver the genes to the cornea. The hydrogels showed adequate light transparency, oxygen permeability, and swelling for use as contact lenses. Loading of viral vectors (rAAV-lacZ, rAAV-RFP, or rAAV-hIGF-I) was carried out at 4 °C to maintain viral vector titer. Release in culture medium was monitored by fluorescence with Cy3-rAAV-lacZ and AAV Titration ELISA. Transduction efficacy was tested through reporter genes lacZ and RFP in human bone marrow derived mesenchymal stem cells (hMSCs). lacZ was detected with X-Gal staining and quantified with Beta-Glo®, and RFP was monitored by fluorescence. The ability of rAAV-hIGF-I-loaded hydrogels to trigger cell proliferation in hMSCs was evaluated by immunohistochemistry. Finally, the ability of rAAV-lacZ-loaded hydrogels to transduce bovine cornea was confirmed through detection with X-Gal staining of β-galactosidase expressed within the tissue. Full article
(This article belongs to the Special Issue Scaffold-Mediated Gene Delivery)
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13 pages, 1750 KiB  
Article
Enhanced Chondrogenic Differentiation Activities in Human Bone Marrow Aspirates via sox9 Overexpression Mediated by pNaSS-Grafted PCL Film-Guided rAAV Gene Transfer
by Jagadeesh K. Venkatesan, Weikun Meng, Ana Rey-Rico, Gertrud Schmitt, Susanne Speicher-Mentges, Céline Falentin-Daudré, Amélie Leroux, Henning Madry, Véronique Migonney and Magali Cucchiarini
Pharmaceutics 2020, 12(3), 280; https://doi.org/10.3390/pharmaceutics12030280 - 21 Mar 2020
Cited by 17 | Viewed by 2788
Abstract
Background: The delivery of therapeutic genes in sites of articular cartilage lesions using non-invasive, scaffold-guided gene therapy procedures is a promising approach to stimulate cartilage repair while protecting the cargos from detrimental immune responses, particularly when targeting chondroreparative bone marrow-derived mesenchymal stromal cells [...] Read more.
Background: The delivery of therapeutic genes in sites of articular cartilage lesions using non-invasive, scaffold-guided gene therapy procedures is a promising approach to stimulate cartilage repair while protecting the cargos from detrimental immune responses, particularly when targeting chondroreparative bone marrow-derived mesenchymal stromal cells in a natural microenvironment like marrow aspirates. Methods: Here, we evaluated the benefits of providing a sequence for the cartilage-specific sex-determining region Y-type high-mobility group box 9 (SOX9) transcription factor to human marrow aspirates via recombinant adeno-associated virus (rAAV) vectors delivered by poly(ε-caprolactone) (PCL) films functionalized via grafting with poly(sodium styrene sulfonate) (pNaSS) to enhance the marrow chondrogenic potential over time. Results: Effective sox9 overexpression was observed in aspirates treated with pNaSS-grafted or ungrafted PCL films coated with the candidate rAAV-FLAG-hsox9 (FLAG-tagged rAAV vector carrying a human sox9 gene sequence) vector for at least 21 days relative to other conditions (pNaSS-grafted and ungrafted PCL films without vector coating). Overexpression of sox9 via rAAV sox9/pNaSS-grafted or ungrafted PCL films led to increased biological and chondrogenic differentiation activities (matrix deposition) in the aspirates while containing premature osteogenesis and hypertrophy without impacting cell proliferation, with more potent effects noted when using pNaSS-grafted films. Conclusions: These findings show the benefits of targeting patients’ bone marrow via PCL film-guided therapeutic rAAV (sox9) delivery as an off-the-shelf system for future strategies to enhance cartilage repair in translational applications. Full article
(This article belongs to the Special Issue Scaffold-Mediated Gene Delivery)
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Review

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23 pages, 1322 KiB  
Review
Scaffold-Mediated Gene Delivery for Osteochondral Repair
by Henning Madry, Jagadeesh Kumar Venkatesan, Natalia Carballo-Pedrares, Ana Rey-Rico and Magali Cucchiarini
Pharmaceutics 2020, 12(10), 930; https://doi.org/10.3390/pharmaceutics12100930 - 29 Sep 2020
Cited by 15 | Viewed by 3277
Abstract
Osteochondral defects involve both the articular cartilage and the underlying subchondral bone. If left untreated, they may lead to osteoarthritis. Advanced biomaterial-guided delivery of gene vectors has recently emerged as an attractive therapeutic concept for osteochondral repair. The goal of this review is [...] Read more.
Osteochondral defects involve both the articular cartilage and the underlying subchondral bone. If left untreated, they may lead to osteoarthritis. Advanced biomaterial-guided delivery of gene vectors has recently emerged as an attractive therapeutic concept for osteochondral repair. The goal of this review is to provide an overview of the variety of biomaterials employed as nonviral or viral gene carriers for osteochondral repair approaches both in vitro and in vivo, including hydrogels, solid scaffolds, and hybrid materials. The data show that a site-specific delivery of therapeutic gene vectors in the context of acellular or cellular strategies allows for a spatial and temporal control of osteochondral neotissue composition in vitro. In vivo, implantation of acellular hydrogels loaded with nonviral or viral vectors has been reported to significantly improve osteochondral repair in translational defect models. These advances support the concept of scaffold-mediated gene delivery for osteochondral repair. Full article
(This article belongs to the Special Issue Scaffold-Mediated Gene Delivery)
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