Design and development of polymers for gene delivery

DW Pack, AS Hoffman, S Pun, PS Stayton - Nature reviews Drug …, 2005 - nature.com
The lack of safe and efficient gene-delivery methods is a limiting obstacle to human gene
therapy. Synthetic gene-delivery agents, although safer than recombinant viruses, generally …

Polymer systems for gene delivery—past, present, and future

SY Wong, JM Pelet, D Putnam - Progress in Polymer Science, 2007 - Elsevier
Gene therapy has long sought to treat disease by delivering therapeutic genes to diseased
cells. However, its widespread clinical use has faltered in the face of several challenges …

Effects of α-synuclein immunization in a mouse model of Parkinson's disease

E Masliah, E Rockenstein, A Adame, M Alford, L Crews… - Neuron, 2005 - cell.com
Abnormal folding of α-synuclein (α-syn) is thought to lead to neurodegeneration and the
characteristic symptoms of Lewy body disease (LBD). Since previous studies suggest that …

Design of multifunctional non-viral gene vectors to overcome physiological barriers: dilemmas and strategies

T Wang, JR Upponi, VP Torchilin - International journal of pharmaceutics, 2012 - Elsevier
Gene-based therapeutics hold great promise for medical advancement and have been used
to treat various human diseases with mixed success. However, their therapeutic application …

shRNA targeting α-synuclein prevents neurodegeneration in a Parkinson's disease model

AD Zharikov, JR Cannon, V Tapias… - The Journal of …, 2015 - Am Soc Clin Investig
Multiple convergent lines of evidence implicate both α-synuclein (encoded by SCNA) and
mitochondrial dysfunction in the pathogenesis of sporadic Parkinson's disease (PD) …

A synopsis on the role of tyrosine hydroxylase in Parkinson's disease

S Tabrez, NR Jabir, S Shakil, NH Greig… - CNS & Neurological …, 2012 - ingentaconnect.com
Parkinson's disease (PD) is a common chronic progressive neurodegenerative disorder in
elderly people. A consistent neurochemical abnormality in PD is degeneration of …

Lipid and hydrophobic modification of cationic carriers on route to superior gene vectors

V Incani, A Lavasanifar, H Uludağ - Soft Matter, 2010 - pubs.rsc.org
Gene therapy has been pursued in the past two decades for treatment of diseases
associated with defective gene expression. The insertion of a therapeutic gene into cells …

Gene delivery to differentiated neurotypic cells with RGD and HIV Tat peptide functionalized polymeric nanoparticles

JS Suk, J Suh, K Choy, SK Lai, J Fu, J Hanes - Biomaterials, 2006 - Elsevier
A number of neurodegenerative disorders may potentially be treated by the delivery of
therapeutic genes to neurons. Nonviral gene delivery systems, however, typically provide …

AAV2-mediated striatum delivery of human CDNF prevents the deterioration of midbrain dopamine neurons in a 6-hydroxydopamine induced parkinsonian rat model

X Ren, T Zhang, X Gong, G Hu, W Ding, X Wang - Experimental neurology, 2013 - Elsevier
Parkinson's disease (PD) is an aging-associated neurodegenerative disorder with
progressive pathology involving the loss of midbrain dopaminergic neurons. Neurotrophic …

Cell penetrating peptide conjugated chitosan for enhanced delivery of nucleic acid

B Layek, L Lipp, J Singh - International journal of molecular sciences, 2015 - mdpi.com
Gene therapy is an emerging therapeutic strategy for the cure or treatment of a spectrum of
genetic disorders. Nevertheless, advances in gene therapy are immensely reliant upon …