First-in-human mutation-targeted siRNA phase Ib trial of an inherited skin disorder. (Q33730680)

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12 February 2020

title

First-in-human mutation-targeted siRNA phase Ib trial of an inherited skin disorder (English)

1 reference

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12 February 2020

4

1 reference

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12 February 2020

Kenneth Boucher

6

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12 February 2020

13

Leonard M Milstone

1 reference

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12 February 2020

author name string

Sancy A Leachman

1

1 reference

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12 February 2020

Robyn P Hickerson

2

1 reference

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12 February 2020

Mary E Schwartz

3

1 reference

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12 February 2020

Stephen L Hutcherson

5

1 reference

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12 February 2020

C David Hansen

7

1 reference

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12 February 2020

Mark J Eliason

8

1 reference

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12 February 2020

G Susan Srivatsa

9

1 reference

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12 February 2020

Douglas J Kornbrust

10

1 reference

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12 February 2020

Frances Jd Smith

11

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12 February 2020

Wh Irwin McLean

12

1 reference

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12 February 2020

Roger L Kaspar

14

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12 February 2020

publication date

24 November 2009

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12 February 2020

1 reference

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12 February 2020

volume

18

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12 February 2020

issue

2

1 reference

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12 February 2020

page(s)

442-446

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siRNA silencing of keratinocyte-specific GFP expression in a transgenic mouse skin model.

12 February 2020

cites work

1 reference

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Knocking down barriers: advances in siRNA delivery

21 June 2018

1 reference

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The promises and pitfalls of RNA-interference-based therapeutics

21 June 2018

1 reference

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Stability study of unmodified siRNA and relevance to clinical use.

21 June 2018

1 reference

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Therapeutic siRNAs for dominant genetic skin disorders including pachyonychia congenita.

21 June 2018

1 reference

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The business of RNAi therapeutics

21 June 2018

1 reference

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RNAi therapeutics: an update on delivery

21 June 2018

1 reference

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Sequence- and target-independent angiogenesis suppression by siRNA via TLR3

21 June 2018

1 reference

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Delivering silence: advancements in developing siRNA therapeutics.

21 June 2018

1 reference

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Single-nucleotide-specific siRNA targeting in a dominant-negative skin model

21 June 2018

1 reference

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Challenges in developing therapies for rare diseases including pachyonychia congenita

21 June 2018

1 reference

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The genetic basis of pachyonychia congenita.

21 June 2018

1 reference

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Clinical and pathological features of pachyonychia congenita

21 June 2018

1 reference

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Overcoming functional redundancy to elicit pachyonychia congenita-like nail lesions in transgenic mice

21 June 2018

1 reference

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An inducible mouse model for epidermolysis bullosa simplex: implications for gene therapy

21 June 2018

1 reference

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Evaluation of the safety, tolerability and pharmacokinetics of ALN-RSV01, a novel RNAi antiviral therapeutic directed against respiratory syncytial virus (RSV).

21 June 2018

1 reference

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30 October 2018

Identifiers

DOI

10.1038/MT.2009.273

1 reference

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12 February 2020

1 reference

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12 February 2020

PubMed publication ID

19935778

1 reference