DNA nanoparticle-mediated ABCA4 delivery rescues Stargardt dystrophy in mice

Direct gene transfer with compacted DNA nanoparticles in retinal pigment epithelial cells: expression, repeat delivery and lack of toxicity

(2012) Zongchao Han et al.   Nanomedicine

Increased cone sensitivity to ABCA4 deficiency provides insight into macular vision loss in Stargardt's dystrophy

(2011) Shannon M. Conley et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE

Compacted DNA Nanoparticle Gene Transfer of GDNF to the Rat Striatum Enhances the Survival of Grafted Fetal Dopamine Neurons

(2010) David M. Yurek et al.   CELL TRANSPLANTATION

Little Vector, Big Gene Transduction: Fragmented Genome Reassembly of Adeno-associated Virus

(2010) Matthew L Hirsch et al.   MOLECULAR THERAPY

Gene delivery to mitotic and postmitotic photoreceptors via compacted DNA nanoparticles results in improved phenotype in a mouse model of retinitis pigmentosa

(2009) Xue Cai et al.   FASEB JOURNAL

Long-term Transgene Expression in the Central Nervous System Using DNA Nanoparticles

(2009) David M Yurek et al.   MOLECULAR THERAPY

A Partial Structural and Functional Rescue of a Retinitis Pigmentosa Model with Compacted DNA Nanoparticles

(2009) Xue Cai et al.   PLoS One

Correction of the disease phenotype in the mouse model of Stargardt disease by lentiviral gene therapy

(2008) J Kong et al.   GENE THERAPY

Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice

(2008) Mariacarmela Allocca et al.   JOURNAL OF CLINICAL INVESTIGATION