Design of a dendrimeric nanomedicine pl atform for the treatment of a neuro-degenerati ve disease, x-ald

Ozgul GoK, Christina L. Nemeth, Bela Turk, Siva Pramodh Kambhampati, Fan Zhang, Anjali Sharma, Sujatha Kannan, Ali Fatemi, Kannan Rangaramanujam

Research output: Chapter in Book/Report/Conference proceedingConference contribution

Abstract

Statement of Purpose: Delivery of therapeutics acros s the blood-brain barrier (BBB) to selectively treat injured brain areas is a major challenge (Kannan, R. M. J Intern Med 2014; 276: 579– 617) and has been a limiting factor for the treat ment of several neurodevelopmental disorders. X-linked adrenoleu kodystrophy (ALD) is a rare genetic dis ease which results in the rapid demyelination of the brain, most severely occurring during childhood. With their great potential as drug/gene delivery nanovehicles, dendrimers provide lots of advantageous features like monodispersity, mu ltivalency, enhanced water solubility and functionalization by imaging agents and targeting groups. We have shown that polyamidoamine (PAMAM) dendrimers can pass through the impaired BBB without any need for additional targeting ligands and localize in the inju red site based on the extent of injury (Zhang, F. J. Neuroin flammation 2016;3-65). In a neonatal rabbit model of cerebral palsy (CP), Cy5-labeled, generation-4 PAMAM dendrimer (D-Cy5) was shown to accumulate almost 20 times more in the brains of rabbit kits with CP, co mpared to healthy controls (Figure 1) (Lesniak, W. G. Mol. Pharmaceutics 2013;10:4560-4571). In light of these findings, a novel PAMAM-based drug delivery system was designed for the delivery of ABCD2 gene regulating drug, 4-phenyl butyrate (PBA), as a brain targeting nano-vehicle with a sustained and slow drug release profile.

Original languageEnglish (US)
Title of host publicationSociety for Biomaterials Annual Meeting and Exposition 2019
Subtitle of host publicationThe Pinnacle of Biomaterials Innovation and Excellence - Transactions of the 42nd Annual Meeting
PublisherSociety for Biomaterials
Number of pages1
ISBN (Electronic)9781510883901
StatePublished - Jan 1 2019
Event42nd Society for Biomaterials Annual Meeting and Exposition 2019: The Pinnacle of Biomaterials Innovation and Excellence - Seattle, United States
Duration: Apr 3 2019Apr 6 2019

Publication series

NameTransactions of the Annual Meeting of the Society for Biomaterials and the Annual International Biomaterials Symposium
Volume40
ISSN (Print)1526-7547

Conference

Conference42nd Society for Biomaterials Annual Meeting and Exposition 2019: The Pinnacle of Biomaterials Innovation and Excellence
CountryUnited States
CitySeattle
Period4/3/194/6/19

ASJC Scopus subject areas

  • Biochemistry
  • Biophysics
  • Biotechnology
  • Biomaterials
  • Materials Chemistry

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  • Cite this

    GoK, O., Nemeth, C. L., Turk, B., Kambhampati, S. P., Zhang, F., Sharma, A., Kannan, S., Fatemi, A., & Rangaramanujam, K. (2019). Design of a dendrimeric nanomedicine pl atform for the treatment of a neuro-degenerati ve disease, x-ald. In Society for Biomaterials Annual Meeting and Exposition 2019: The Pinnacle of Biomaterials Innovation and Excellence - Transactions of the 42nd Annual Meeting (Transactions of the Annual Meeting of the Society for Biomaterials and the Annual International Biomaterials Symposium; Vol. 40). Society for Biomaterials.