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Items: 1 to 20 of 96

1.

Efficient and Robust Highly Branched Poly(β-amino ester)/Minicircle COL7A1 Polymeric Nanoparticles for Gene Delivery to Recessive Dystrophic Epidermolysis Bullosa Keratinocytes.

Zeng M, Alshehri F, Zhou D, Lara-Sáez I, Wang X, Li X, A S, Xu Q, Zhang J, Wang W.

ACS Appl Mater Interfaces. 2019 Aug 28;11(34):30661-30672. doi: 10.1021/acsami.9b13135. Epub 2019 Aug 20.

PMID:
31390173
2.

Manipulation of Transgene Expression in Fibroblast Cells by a Multifunctional Linear-Branched Hybrid Poly(β-Amino Ester) Synthesized through an Oligomer Combination Approach.

Zeng M, Zhou D, Alshehri F, Lara-Sáez I, Lyu Y, Creagh-Flynn J, Xu Q, A S, Zhang J, Wang W.

Nano Lett. 2019 Jan 9;19(1):381-391. doi: 10.1021/acs.nanolett.8b04098. Epub 2018 Dec 21.

PMID:
30565945
3.

The transition from linear to highly branched poly(β-amino ester)s: Branching matters for gene delivery.

Zhou D, Cutlar L, Gao Y, Wang W, O'Keeffe-Ahern J, McMahon S, Duarte B, Larcher F, Rodriguez BJ, Greiser U, Wang W.

Sci Adv. 2016 Jun 17;2(6):e1600102. doi: 10.1126/sciadv.1600102. eCollection 2016 Jun.

4.

Highly Branched poly(5-amino-1-pentanol-co-1,4-butanediol diacrylate) for High Performance Gene Transfection.

Zeng M, Zhou D, Ng S, Ahern JOK, Alshehri F, Gao Y, Pierucci L, Greiser U, Wang W.

Polymers (Basel). 2017 May 1;9(5). pii: E161. doi: 10.3390/polym9050161.

5.

Keratinocyte-/fibroblast-targeted rescue of Col7a1-disrupted mice and generation of an exact dystrophic epidermolysis bullosa model using a human COL7A1 mutation.

Ito K, Sawamura D, Goto M, Nakamura H, Nishie W, Sakai K, Natsuga K, Shinkuma S, Shibaki A, Uitto J, Denton CP, Nakajima O, Akiyama M, Shimizu H.

Am J Pathol. 2009 Dec;175(6):2508-17. doi: 10.2353/ajpath.2009.090347. Epub 2009 Nov 5.

6.

Highly branched poly(β-amino ester)s for skin gene therapy.

Zhou D, Gao Y, Aied A, Cutlar L, Igoucheva O, Newland B, Alexeeve V, Greiser U, Uitto J, Wang W.

J Control Release. 2016 Dec 28;244(Pt B):336-346. doi: 10.1016/j.jconrel.2016.06.014. Epub 2016 Jun 8.

PMID:
27288877
7.

High levels of type VII collagen expression in recessive dystrophic epidermolysis bullosa cutaneous squamous cell carcinoma keratinocytes increases PI3K and MAPK signalling, cell migration and invasion.

Pourreyron C, Chen M, McGrath JA, Salas-Alanis JC, South AP, Leigh IM.

Br J Dermatol. 2014 Jun;170(6):1256-65. doi: 10.1111/bjd.12715.

PMID:
24641191
8.

Correction of Recessive Dystrophic Epidermolysis Bullosa by Transposon-Mediated Integration of COL7A1 in Transplantable Patient-Derived Primary Keratinocytes.

Latella MC, Cocchiarella F, De Rosa L, Turchiano G, Gonçalves MAFV, Larcher F, De Luca M, Recchia A.

J Invest Dermatol. 2017 Apr;137(4):836-844. doi: 10.1016/j.jid.2016.11.038. Epub 2016 Dec 24.

9.

Targeted Exon Skipping Restores Type VII Collagen Expression and Anchoring Fibril Formation in an In Vivo RDEB Model.

Turczynski S, Titeux M, Tonasso L, Décha A, Ishida-Yamamoto A, Hovnanian A.

J Invest Dermatol. 2016 Dec;136(12):2387-2395. doi: 10.1016/j.jid.2016.07.029. Epub 2016 Aug 3.

10.

A knot polymer mediated non-viral gene transfection for skin cells.

Cutlar L, Gao Y, Aied A, Greiser U, Murauer EM, Zhou D, Wang W.

Biomater Sci. 2016 Jan;4(1):92-5. doi: 10.1039/c5bm00216h.

PMID:
26369723
11.

Ex Vivo COL7A1 Correction for Recessive Dystrophic Epidermolysis Bullosa Using CRISPR/Cas9 and Homology-Directed Repair.

Izmiryan A, Ganier C, Bovolenta M, Schmitt A, Mavilio F, Hovnanian A.

Mol Ther Nucleic Acids. 2018 Sep 7;12:554-567. doi: 10.1016/j.omtn.2018.06.008. Epub 2018 Jun 26.

12.

Safety and Wound Outcomes Following Genetically Corrected Autologous Epidermal Grafts in Patients With Recessive Dystrophic Epidermolysis Bullosa.

Siprashvili Z, Nguyen NT, Gorell ES, Loutit K, Khuu P, Furukawa LK, Lorenz HP, Leung TH, Keene DR, Rieger KE, Khavari P, Lane AT, Tang JY, Marinkovich MP.

JAMA. 2016 Nov 1;316(17):1808-1817. doi: 10.1001/jama.2016.15588.

PMID:
27802546
13.

Functional correction of type VII collagen expression in dystrophic epidermolysis bullosa.

Murauer EM, Gache Y, Gratz IK, Klausegger A, Muss W, Gruber C, Meneguzzi G, Hintner H, Bauer JW.

J Invest Dermatol. 2011 Jan;131(1):74-83. doi: 10.1038/jid.2010.249. Epub 2010 Aug 19.

14.

Aminoglycosides restore full-length type VII collagen by overcoming premature termination codons: therapeutic implications for dystrophic epidermolysis bullosa.

Cogan J, Weinstein J, Wang X, Hou Y, Martin S, South AP, Woodley DT, Chen M.

Mol Ther. 2014 Oct;22(10):1741-52. doi: 10.1038/mt.2014.140. Epub 2014 Jul 23.

15.

Restoration of type VII collagen expression and function in dystrophic epidermolysis bullosa.

Chen M, Kasahara N, Keene DR, Chan L, Hoeffler WK, Finlay D, Barcova M, Cannon PM, Mazurek C, Woodley DT.

Nat Genet. 2002 Dec;32(4):670-5. Epub 2002 Nov 11.

PMID:
12426566
16.

Amlexanox Enhances Premature Termination Codon Read-Through in COL7A1 and Expression of Full Length Type VII Collagen: Potential Therapy for Recessive Dystrophic Epidermolysis Bullosa.

Atanasova VS, Jiang Q, Prisco M, Gruber C, Piñón Hofbauer J, Chen M, Has C, Bruckner-Tuderman L, McGrath JA, Uitto J, South AP.

J Invest Dermatol. 2017 Sep;137(9):1842-1849. doi: 10.1016/j.jid.2017.05.011. Epub 2017 May 24. Review.

17.

Gene Editing for the Efficient Correction of a Recurrent COL7A1 Mutation in Recessive Dystrophic Epidermolysis Bullosa Keratinocytes.

Chamorro C, Mencía A, Almarza D, Duarte B, Büning H, Sallach J, Hausser I, Del Río M, Larcher F, Murillas R.

Mol Ther Nucleic Acids. 2016 Apr 5;5:e307. doi: 10.1038/mtna.2016.19.

18.

Recurrent nonsense mutations within the type VII collagen gene in patients with severe recessive dystrophic epidermolysis bullosa.

Hovnanian A, Hilal L, Blanchet-Bardon C, de Prost Y, Christiano AM, Uitto J, Goossens M.

Am J Hum Genet. 1994 Aug;55(2):289-96.

19.

Construction of skin equivalents for gene therapy of recessive dystrophic epidermolysis bullosa.

Gache Y, Baldeschi C, Del Rio M, Gagnoux-Palacios L, Larcher F, Lacour JP, Meneguzzi G.

Hum Gene Ther. 2004 Oct;15(10):921-33.

PMID:
15585108
20.

Recessive dystrophic epidermolysis bullosa caused by COL7A1 hemizygosity and a missense mutation with complex effects on splicing.

Titeux M, Mejía JE, Mejlumian L, Bourthoumieu S, Mirval S, Tonasso L, Heller M, Prost-Squarcioni C, Hovnanian A.

Hum Mutat. 2006 Mar;27(3):291-2.

PMID:
16470588

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