Delivery of Therapeutic Proteins via Extracellular Vesicles: Review and Potential Treatments for Parkinson’s Disease, Glioma, and Schwannoma

Hall, Justin S.; Prabhakar, Shilpa; Balaj, Leonora; Lai, Charles P.; Cerione, Richard A.; Breakefield, Xandra O.

doi:10.1007/s10571-015-0309-0

Cited by 96 publications

(92 citation statements)

References 77 publications

(89 reference statements)

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“…In the case of NF2, an AAV vector encoding a cell death gene, caspase-1 under a Schwann cell specific promoter has been effective at reducing the growth and leading to regression of human schwannoma tumors implanted in the sciatic nerve of nude mice without causing nerve damage (Prabhakar et al, 2013), and may have a bystander effect via extracellular vesicles (Hall et al, in press). This targeted cell death approach has the advantage that it could be applied to a variety of benign tumor types accessible to injection by using different tumor-specific promoters.…”

Section: Different Modalities For Different Diseasesmentioning

confidence: 99%

Viral vectors for therapy of neurologic diseases

et al. 2017

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Neurological disorders – disorders of the brain, spine and associated nerves – are a leading contributor to global disease burden with a shockingly large associated economic cost. Various treatment approaches – pharmaceutical medication, device-based therapy, physiotherapy, surgical intervention, among others – have been explored to alleviate the resulting extent of human suffering. In recent years, gene therapy using viral vectors – encoding a therapeutic gene or inhibitory RNA into a “gutted” viral capsid and supplying it to the nervous system – has emerged as a clinically viable option for therapy of brain disorders. In this Review, we provide an overview of the current state and advances in the field of viral vector-mediated gene therapy for neurological disorders. Vector tools and delivery methods have evolved considerably over recent years, with the goal of providing greater and safer genetic access to the central nervous system. Better etiological understanding of brain disorders has concurrently led to identification of improved therapeutic targets. We focus on the vector technology, as well as preclinical and clinical progress made thus far for brain cancer and various neurodegenerative and neurometabolic disorders, and point out the challenges and limitations that accompany this new medical modality. Finally, we explore the directions that neurological gene therapy is likely to evolve towards in the future.

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Section: Different Modalities For Different Diseasesmentioning

confidence: 99%

Viral vectors for therapy of neurologic diseases

et al. 2017

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“…EVs have the potential to serve as a next-generation drug vehicle owing to their low immunogenicity, tissue targeting capability, ability to evade phagocytic clearance, and stability in circulation [12]. Because of their small size, exosomes take advantage of the enhanced permeability and retention effect for tumor targeting.…”

Section: Types Of Circulatory Cells In Drug Delivery Applicationsmentioning

confidence: 99%

Next generation drug delivery: circulatory cells-mediated nanotherapeutic approaches

Agrahari

Mitra

2016

Expert Opinion on Drug Delivery

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“…Besides anti-cancer therapeutic drugs, exosomes can also deliver various tumor antigens (Cho et al, 2005), apoptosis-inducing proteins (Hall et al, 2016), nanobodies (Kooijmans et al, 2016), deficient or mutant anti-apoptosis proteins (Aspe et al, 2014), tumor and tissue-specific peptides (Hung and Leonard, 2015), proteasomes (Lai et al, 2012), transferrins, and lactoferrins (Malhotra et al, 2016) into cancer cells for targeting therapy.…”

Section: Introductionmentioning

confidence: 99%

Exosome-Based Cancer Therapy: Implication for Targeting Cancer Stem Cells

2017

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Drug resistance, difficulty in specific targeting and self-renewal properties of cancer stem cells (CSCs) all contribute to cancer treatment failure and relapse. CSCs have been suggested as both the seeds of the primary cancer, and the roots of chemo- and radio-therapy resistance. The ability to precisely deliver drugs to target CSCs is an urgent need for cancer therapy, with nanotechnology-based drug delivery system being one of the most promising tools to achieve this in the clinic. Exosomes are cell-derived natural nanometric vesicles that are widely distributed in body fluids and involved in multiple disease processes, including tumorigenesis. Exosome-based nanometric vehicles have a number of advantages: they are non-toxic, non-immunogenic, and can be engineered to have robust delivery capacity and targeting specificity. This enables exosomes as a powerful nanocarrier to deliver anti-cancer drugs and genes for CSC targeting therapy. Here, we will introduce the current explorations of exosome-based delivery system in cancer therapy, with particular focus on several exosomal engineering approaches that have improved their efficiency and specificity for CSC targeting.

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Delivery of Therapeutic Proteins via Extracellular Vesicles: Review and Potential Treatments for Parkinson’s Disease, Glioma, and Schwannoma

Cited by 96 publications

References 77 publications

Viral vectors for therapy of neurologic diseases

Viral vectors for therapy of neurologic diseases

Next generation drug delivery: circulatory cells-mediated nanotherapeutic approaches

Exosome-Based Cancer Therapy: Implication for Targeting Cancer Stem Cells

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