RNAi-mediated silencing of hepatic Alas1 effectively prevents and treats the induced acute attacks in acute intermittent porphyria mice (Q33694417)

28 July 2017

RNAi-mediated silencing of hepatic Alas1 effectively prevents and treats the induced acute attacks in acute intermittent porphyria mice (English)

1 reference

acute intermittent porphyria

28 July 2017

1 reference

5

Chunli Yu

1 reference

28 July 2017

11

Robert J Desnick

1 reference

28 July 2017

Makiko Yasuda

1

1 reference

28 July 2017

Lin Gan

2

1 reference

28 July 2017

Brenden Chen

3

1 reference

28 July 2017

Senkottuvelan Kadirvel

4

1 reference

28 July 2017

John D Phillips

6

1 reference

28 July 2017

Maria I New

7

1 reference

28 July 2017

Abigail Liebow

8

1 reference

28 July 2017

Kevin Fitzgerald

9

1 reference

28 July 2017

William Querbes

10

1 reference

28 July 2017

language of work or name

English

0 references

publication date

12 May 2014

1 reference

Proceedings of the National Academy of Sciences of the United States of America

28 July 2017

1 reference

28 July 2017

111

1 reference

28 July 2017

21

1 reference

28 July 2017

7777-7782

1 reference

Effect of an RNA interference drug on the synthesis of proprotein convertase subtilisin/kexin type 9 (PCSK9) and the concentration of serum LDL cholesterol in healthy volunteers: a randomised, single-blind, placebo-controlled, phase 1 trial

28 July 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4040563

Treatment of HCV infection by targeting microRNA

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4040563

First-in-humans trial of an RNA interference therapeutic targeting VEGF and KSP in cancer patients with liver involvement

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4040563

Maximizing the potency of siRNA lipid nanoparticles for hepatic gene silencing in vivo

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4040563

A LC-MS/MS method for the specific, sensitive, and simultaneous quantification of 5-aminolevulinic acid and porphobilinogen

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4040563

Sustained enzymatic correction by rAAV-mediated liver gene therapy protects against induced motor neuropathy in acute porphyria mice.

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4040563

Targeted delivery of RNAi therapeutics with endogenous and exogenous ligand-based mechanisms

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4040563

Therapeutic RNAi targeting PCSK9 acutely lowers plasma cholesterol in rodents and LDL cholesterol in nonhuman primates

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4040563

Development and properties of HepG2 cells that constitutively express CYP2E1.

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4040563

Liver transplantation for porphyria: who, when, and how?

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4040563

Recommendations for the diagnosis and treatment of the acute porphyrias

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4040563

Adenoviral-mediated expression of porphobilinogen deaminase in liver restores the metabolic defect in a mouse model of acute intermittent porphyria

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4040563

Recovery from a variegate porphyria by a liver transplantation

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4040563

Liver transplantation as a cure for acute intermittent porphyria

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4040563

The activation and induction of tryptophan pyrrolase during experimental porphyria and by amino-triazole.

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4040563

Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4040563

Acute porphyrias: pathogenesis of neurological manifestations

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4040563

Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4040563

Regulation by heme of mitochondrial protein transport through a conserved amino acid motif

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4040563

Early administration of heme arginate for acute porphyric attacks

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4040563

Repression of the Overproduction of Porphyrin Precursors in Acute Intermittent Porphyria by Intravenous Infusions of Hematin

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4040563

Disturbances of liver porphyrin metabolism caused by drugs

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4040563

Structure, turnover, and heme-mediated suppression of the level of mRNA encoding rat liver delta-aminolevulinate synthase

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4040563

Haem arginate in the treatment of acute hepatic porphyrias

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4040563

Regulation of the stability of chicken embryo liver delta-aminolevulinate synthase mRNA by hemin

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4040563

Tryptophan pyrrolase, the regulatory free haem and hepatic porphyrias. Early depletion of haem by clinical and experimental exacerbators of porphyria

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4040563

Steroid induction of delta-aminolevulinic acid synthase and porphyrins in liver. Structure-activity studies and the permissive effects of hormones on the induction process

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4040563

Nutritional regulation of hepatic heme biosynthesis and porphyria through PGC-1alpha

21 June 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.1406228111

RNAi-mediated gene silencing in non-human primates.

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.1406228111

Porphobilinogen deaminase deficiency in mice causes a neuropathy resembling that of human hepatic porphyria

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.1406228111

Safety and efficacy of RNAi therapy for transthyretin amyloidosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.1406228111

Dyslipidaemia: RNAi targeting PCSK9 decreases lipid levels in a human trial.

21 January 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4040563

Assays of the heme biosynthetic enzymes. Preface

30 October 2018

1 reference

12 December 2020

The effect of progesterone on activities of delta-aminolevulinic acid synthetase and delta-aminolevulinic acid dehydratase in estrogen-primed avian oviduct

1 reference

12 December 2020

Liver Transplantation From Donors With Acute Intermittent Porphyria

1 reference

12 December 2020

Induction of the synthesis of delta-aminolevulinic acid synthetase in liver parenchyma cells in culture by chemical that induce acute porphyria

1 reference

12 December 2020

Instability of hematin used in the treatment of acute hepatic porphyria

1 reference

12 December 2020

Assessment of arsenic effects on cytosolic heme status using tryptophan pyrrolase as an index

1 reference

12 December 2020

Identifiers

DOI

10.1073/PNAS.1406228111

1 reference

28 July 2017

0 references

1 reference

28 July 2017

1 reference