Neuroprotective Gene Therapy for Huntington’s Disease Using a Polymer Encapsulated BHK Cell Line Engineered to Secrete Human CNTF

Bachoud‐Lévi, Anne‐Catherine; Déglon, Nicole; Nǵuyen, Jean-Paul; Bloch, Jocelyne; Bourdet, Catherine; Winkel, L.; Rémy, Philippe; Goddard, Moses; Lefaucheur, J.-P.; Brugières, Pierre; Baudic, Sophie; Césaro, P.; Peschanski, Marc; Aebischer, Patrick

doi:10.1089/10430340050111377

Cited by 152 publications

(63 citation statements)

References 53 publications

Supporting

Mentioning

Contrasting

Unclassified

Order By: Relevance

“…Cells can also serve as production factories to produce and correctly process these agents in their most physiological form. In this regard, genetically engineered cells have been used to produce and secrete a wide range of transgene products including factor IX, 3 growth hormone, 4 erythropoietin, 5 ciliary neurotrophic factor 6 and dopamine. 7 Increased utilization of cells as vehicles has helped to identify key factors that are critical and distinct for this method of therapeutic delivery.…”

Section: Cells Have Distinct Advantages As Carriers Of Complex Therapmentioning

confidence: 99%

Cell vehicle targeting strategies

2008

View full text Add to dashboard Cite

Use of cells as therapeutic carriers has increased in the past few years and has developed as a distinct concept and delivery method. Cell-based vehicles are particularly attractive for delivery of biotherapeutic agents that are difficult to synthesize, have reduced half-lives, limited tissue penetrance or are rapidly inactivated upon direct in vivo introduction. Initial studies using cell-based approaches served to identify some of the key factors for the success of this type of therapeutic delivery. These factors include the efficiency of cell loading with a therapeutic payload, the means of cell loading and the nature of therapeutics that cells can carry. However, one important aspect of cell-based delivery yet to be fully investigated is the process of actual delivery of the cell payload in vivo. In this regard, the potential ability of cell carriers to provide site-specific or targeted delivery of therapeutics deserves special attention. The present review focuses on a variety of targeting approaches that may be utilized to improve cell-based therapeutic delivery strategies. The different aspects of targeting that can be applied to cell vehicles will be discussed, including physical methods for directing cell distribution, intrinsic cell-mediated homing mechanisms and the feasibility of engineering cells with novel targeting mechanisms. Development of cell targeting strategies will further advance cell vehicle applications, broaden the applicability of this delivery approach and potentiate therapeutic outcomes.

show abstract

Section: Cells Have Distinct Advantages As Carriers Of Complex Therapmentioning

confidence: 99%

Cell vehicle targeting strategies

2008

View full text Add to dashboard Cite

show abstract

“…36 The use of the semipermeable capsules, implanted in the CNS or cerebral ventricles, allow xenografts and indirect gene therapy with an acceptable level of security. 37 Most of the trials for PD used heterotopic injections; some evidence now indicates that homotopic grafts or cograft may be possible or preferable. 38 …”

Section: Graft Proceduresmentioning

confidence: 99%

The design of clinical trials for cell transplantation into the central nervous system

Césaro

2004

Neurotherapeutics

View full text Add to dashboard Cite

“…So far, encapsulation has been used primarily to treat T1DM [5][6][7][8][9]. By providing a physical barrier to immune rejection, islet encapsulation has been shown to allow transplanted islet to function normally and avoid the use of immunosuppression [10,11].…”

Section: Introductionmentioning

confidence: 99%

Avoiding Immunosuppression for Islet Transplantation: Use of Protective Biomaterials

Alexander¹,

Nguyen²,

Flores³

et al. 2017

Challenges in Pancreatic Pathology

View full text Add to dashboard Cite

Islet transplantation, with the advent of the Edmonton protocol in 2000, has offered a significant alternative for long-lasting treatment of type 1 diabetes. However, the immunosuppression required for transplantation has the cytotoxic effect on pancreatic islets, and thus limiting the long-term efficacy of the transplant. Immediate loss of islets after transplant was also observed because of immediate blood-mediated inflammatory response (IBMIR), which kills islets transplanted in the liver through portal vein. There is also commonly a lack of microvascular blood supply to the transplanted islets. In this chapter, we will review the variety of technologies used to protect transplanted islets against toxicity of immunosuppression, immune rejection, and inflammatory response. We will evaluate the mechanisms of these technologies and their progress in solving the challenges to islet transplantation. The technologies include encapsulation of transplanted islets in various polymers, transplants in sites other than the liver, and creation of new prevascularized transplant site. These technologies offer several mechanisms to prevent immune rejection or immediate contact with cytotoxic inflammatory response, in addition to maintaining islet integrity. New transplant sites are also being developed to support the islets, by allowing establishment of microvasculature and innervation, prior to addition of the islets.

show abstract

Neuroprotective Gene Therapy for Huntington’s Disease Using a Polymer Encapsulated BHK Cell Line Engineered to Secrete Human CNTF

Cited by 152 publications

References 53 publications

Cell vehicle targeting strategies

Cell vehicle targeting strategies

The design of clinical trials for cell transplantation into the central nervous system

Avoiding Immunosuppression for Islet Transplantation: Use of Protective Biomaterials

Contact Info

Product

Resources

About