Transplantation of Cells in an lmmunoisolation Device for Gene Therapy

Carr-Brendel, Victoria; Geller, Robin L.; Thomas, Tracy J.; Boggs, Daniel R.; Young, S. K.; Crudele, Joanne; Martinson, Laura A.; Maryanov, David A.; Johnson, Robert C.; Brauker, James H.

doi:10.1385/0-89603-481-x:373

Cited by 8 publications

(9 citation statements)

References 3 publications

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“…8 In addition, ePTFE has shown promise for use in immunoisolation devices. 14,16 Immuno-isolation devices are intended to immunologically isolate foreign tissue (such as transplanted pancreatic islet tissue) from the host while sustaining the metabolic needs of the encapsulated cells. As such, the combination of minimal fibrosis and enhanced neovascularization near the material is required.…”

Section: Discussionmentioning

confidence: 99%

Evaluation of an aqueous drainage glaucoma device constructed of ePTFE

Boswell¹,

Noecker

Mac

et al. 1999

J. Biomed. Mater. Res.

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Aqueous drainage devices for the treatment of glaucoma are subject to the same limitations as most polymeric implants, namely a healing response comprised of chronic inflammation and fibrosis. The most widely used devices are currently made of silicone or polypropylene, materials that exhibit biocompatibility difficulties when they are implanted on the sclera underneath the conjunctiva of the eye. Decreased outflow of aqueous fluid to the conjunctival space caused by the development of a fibrous capsule around the device accounts for at least 20% of aqueous shunts failures. Clearly, the need exists to improve the healing response to aqueous drainage devices, and one approach is to develop new polymers or polymer modifications. Improved devices would elicit a limited fibrotic response while increasing neovascularization around the implant. Previous studies have indicated that denucleation markedly improves the healing characteristics and biocompatibility of expanded polytetrafluoroethylene (ePTFE). We reasoned that altering the design of drainage devices to allow the use of denucleated ePTFE in vivo might minimize fibrosis, thereby improving shunt function. We found that after 8 weeks in vivo, experimental shunt function was equivalent to the Baerveldt shunt, while there was less scarring with increased neovascularizatin. These findings suggest that ePTFE has potential as an improved, long-term alternative material for use in constructing glaucoma shunts.

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Section: Discussionmentioning

confidence: 99%

Evaluation of an aqueous drainage glaucoma device constructed of ePTFE

Boswell¹,

Noecker

Mac

et al. 1999

J. Biomed. Mater. Res.

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“…It is well documented that transfected cells may harbor plasmid DNA for a fairly long time without detectable transgene expression. 79 A judicious vector design is critical to achieve sustained transgene expression. Improvements in vector design using tissuespecific genetic regulatory elements that affect transgene expression and stability resulted in higher and more stable transgene expression.…”

Section: Vector Optimizationmentioning

confidence: 99%

Nonviral Gene Therapy Approaches to Hemophilia

Gomez-Vargas

Hortelano²

2004

Semin Thromb Hemost

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The goal of hemophilia gene therapy is to obtain long-term therapeutic levels of factor VIII (FVIII) or factor IX (FIX) without stimulating an immune response against the transgene product or the vector. The success of gene therapy is largely dependent on the development of appropriate gene delivery vectors. Both viral vectors and nonviral vectors have been considered for the development of hemophilia gene therapy. In general, viral vectors are far more efficient than nonviral gene delivery approaches and resulted in long-term therapeutic levels of FVIII or FIX in preclinical animal models. However, there are several reasons why a nonviral treatment would still be desirable, particularly because some viral vectors are associated with inflammatory reactions, that render transgene expression transient, or with an increased risk of insertional oncogenesis when random integrating vectors are used. Nonviral vectors may obviate some of these concerns. Since nonviral vectors are typically assembled in cell-free systems from well-defined components, they have significant manufacturing advantages over viral vectors. The continued development of improved nonviral gene delivery approaches offers new perspectives for gene therapy of chronic diseases including hemophilia.

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“…Recently, TheraCyte implantation bags were used in vivo for delivery of Human Factor IX, antitumor antigen, recombinant human growth hormone and insulin (128)(129)(130)132,133). We initiated a series of in vitro experiments to test Theracyte implantation bags as a tool for gene delivery by retroviral vectors.…”

Section: In Vivo Cell-type-specific Gene Deliverymentioning

confidence: 99%

Reticuloendotheliosis viruses and derived vectors for human gene therapy

Dornburg

2003

Front Biosci

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The reticuloendotheliosis viruses (REV) spleen necrosis virus (SNV) and reticuloendotheliosis virus strain-A (REV-A) are amphotropic retroviruses which infect a large variety of cells of avian and some mammalian species. They normally do not infect primate or rodent cells. However, they efficiently infect and integrate their genome into that of human cells when they are pseudotyped with the envelope protein of other mammalian retroviruses or the G protein of vesicular stomatitis virus (VSV) or rabies viruses (RV). Moreover, SNV-derived retroviral vectors, which display single chain antibodies or other targeting ligands on the viral surface enable cell-type-specific gene delivery into various human cells. My laboratory has developed genetically engineered REV vectors, which are capable of infecting non-dividing cells such as quiescent human T-cells, primary monocyte-derived macrophages, and mature neurons. Thus, REV-derived vectors appear to be very interesting candidates for the further development of vectors for human gene therapy. This article reviews the replication of REVs and vectors derived from REV-A and SNV for gene transfer into human cells.

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Transplantation of Cells in an lmmunoisolation Device for Gene Therapy

Cited by 8 publications

References 3 publications

Evaluation of an aqueous drainage glaucoma device constructed of ePTFE

Evaluation of an aqueous drainage glaucoma device constructed of ePTFE

Nonviral Gene Therapy Approaches to Hemophilia

Reticuloendotheliosis viruses and derived vectors for human gene therapy

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