S/MAR-containing DNA nanoparticles promote persistent RPE gene expression and improvement in RPE65-associated LCA (Q36709143)

15 August 2017

S/MAR-containing DNA nanoparticles promote persistent RPE gene expression and improvement in RPE65-associated LCA (English)

1 reference

15 August 2017

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Adarsha Koirala

1

1 reference

15 August 2017

Rasha S Makkia

2

1 reference

15 August 2017

Shannon M Conley

3

1 reference

15 August 2017

Mark J Cooper

4

1 reference

15 August 2017

Muna I Naash

5

1 reference

15 August 2017

18 January 2013

1 reference

15 August 2017

Human Molecular Genetics

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15 August 2017

22

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15 August 2017

8

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15 August 2017

1632-1642

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describes a project that uses

15 August 2017

ImageJ

1 reference

11 June 2022

https://www.ebi.ac.uk/europepmc/webservices/rest/PMC3605833/fullTextXML

based on heuristic

inferred from PubMed Central ID database lookup

cites work

DNA nanoparticle-mediated ABCA4 delivery rescues Stargardt dystrophy in mice.

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

30 September 2017

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Optimization of hCFTR lung expression in mice using DNA nanoparticles

30 September 2017

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Nanoparticle-mediated gene transfer specific to retinal pigment epithelial cells

30 September 2017

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DNA nanoparticles: detection of long-term transgene activity in brain using bioluminescence imaging

30 September 2017

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Gene therapy rescues cone structure and function in the 3-month-old rd12 mouse: a model for midcourse RPE65 leber congenital amaurosis

30 September 2017

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Nucleolin-Mediated Cellular Trafficking of DNA Nanoparticle Is Lipid Raft and Microtubule Dependent and Can Be Modulated by Glucocorticoid

30 September 2017

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Differences in RDS trafficking, assembly and function in cones versus rods: insights from studies of C150S-RDS

30 September 2017

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Leber congenital amaurosis due to RPE65 mutations and its treatment with gene therapy

30 September 2017

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Bestrophins and retinopathies

30 September 2017

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

30 September 2017

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Effect of genome size on AAV vector packaging

30 September 2017

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Ocular delivery of compacted DNA-nanoparticles does not elicit toxicity in the mouse retina

30 September 2017

1 reference

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Compacted DNA nanoparticle gene transfer of GDNF to the rat striatum enhances the survival of grafted fetal dopamine neurons

30 September 2017

1 reference

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Evaluation of 9-cis-retinyl acetate therapy in Rpe65-/- mice

30 September 2017

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A partial structural and functional rescue of a retinitis pigmentosa model with compacted DNA nanoparticles

30 September 2017

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Subretinal delivery of adeno-associated virus serotype 2 results in minimal immune responses that allow repeat vector administration in immunocompetent mice.

30 September 2017

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Long-term transgene expression in the central nervous system using DNA nanoparticles

30 September 2017

1 reference

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Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics

30 September 2017

1 reference

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Effect of gene therapy on visual function in Leber's congenital amaurosis

30 September 2017

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Safety and efficacy of gene transfer for Leber's congenital amaurosis

30 September 2017

1 reference

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Efficient non-viral ocular gene transfer with compacted DNA nanoparticles

30 September 2017

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Lentiviral gene transfer of RPE65 rescues survival and function of cones in a mouse model of Leber congenital amaurosis

30 September 2017

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Diseases caused by defects in the visual cycle: retinoids as potential therapeutic agents

30 September 2017

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Designing nonviral vectors for efficient gene transfer and long-term gene expression

30 September 2017

1 reference

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Plasmid size up to 20 kbp does not limit effective in vivo lung gene transfer using compacted DNA nanoparticles

30 September 2017

1 reference

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Pharmacological and rAAV gene therapy rescue of visual functions in a blind mouse model of Leber congenital amaurosis.

30 September 2017

1 reference

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Gene transfer into human hematopoietic progenitor cells with an episomal vector carrying an S/MAR element

30 September 2017

1 reference

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The nonviral episomal replicating vector pEPI-1 allows long-term inhibition of bcr-abl expression by shRNA.

30 September 2017

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Downregulation of cone-specific gene expression and degeneration of cone photoreceptors in the Rpe65-/- mouse at early ages

30 September 2017

1 reference

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Recombinant adeno-associated virus type 2-mediated gene delivery into the Rpe65-/- knockout mouse eye results in limited rescue

30 September 2017

1 reference

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Chromatin loops are selectively anchored using scaffold/matrix-attachment regions

30 September 2017

1 reference

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GRK1-dependent phosphorylation of S and M opsins and their binding to cone arrestin during cone phototransduction in the mouse retina

30 September 2017

1 reference

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Transcriptional augmentation: modulation of gene expression by scaffold/matrix-attached regions (S/MAR elements).

30 September 2017

1 reference

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Rat retinal pigment epithelial cells show specificity of phagocytosis in vitro

30 September 2017

1 reference

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Transfection of airway epithelium by stable PEGylated poly-L-lysine DNA nanoparticles in vivo

30 September 2017

1 reference

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Nanoparticles of compacted DNA transfect postmitotic cells

28 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3605833

An episomally replicating vector binds to the nuclear matrix protein SAF-A in vivo

28 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3605833

Ultraviolet and middle wavelength sensitive cone responses in the electroretinogram (ERG) of normal and Rpe65 -/- mice

28 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3605833

Ultrastructure of age changes in the basal infoldings of aged mouse retinal pigment epithelium

28 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3605833

Non-viral S/MAR vectors replicate episomally in vivo when provided with a selective advantage

28 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3605833

Cell cycle dependent histone dynamics of an episomal non-viral vector

5 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3605833

RPE65 gene delivery restores isomerohydrolase activity and prevents early cone loss in Rpe65-/- mice

5 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3605833

Retinal degeneration 12 (rd12): a new, spontaneously arising mouse model for human Leber congenital amaurosis (LCA).

5 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3605833

In utero gene therapy rescues vision in a murine model of congenital blindness

5 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3605833

Minimal toxicity of stabilized compacted DNA nanoparticles in the murine lung

5 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3605833

Exploiting a minimal system to study the epigenetic control of DNA replication: the interplay between transcription and replication.

5 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3605833

Rpe65 is necessary for production of 11-cis-vitamin A in the retinal visual cycle.

5 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3605833

Cell surface nucleolin serves as receptor for DNA nanoparticles composed of pegylated polylysine and DNA

5 September 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/23335596

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1093/HMG/DDT013

1 reference

15 August 2017

1 reference

15 August 2017

PubMed publication ID

23335596

1 reference