(Q38931024)

7 September 2017

1 reference

title

Precision medicine, genomics and drug discovery (English)

1 reference

7 September 2017

0 references

1 reference

Lon R Cardon

1

1 reference

7 September 2017

Tim Harris

series ordinal

2

1 reference

7 September 2017

publication date

18 August 2016

1 reference

7 September 2017

published in

Human Molecular Genetics

1 reference

Treating Immunodeficiency through HSC Gene Therapy

7 September 2017

cites work

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Making Better Chimeric Antigen Receptors for Adoptive T-cell Therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

A pharmacologic perspective on newly emerging T-cell manipulation technologies

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Development of immuno-oncology drugs - from CTLA4 to PD1 to the next generations.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

A new initiative on precision medicine

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Personalized medicine: hope or hype?

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Implementing genomic medicine in the clinic: the future is here

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Rare-disease genetics in the era of next-generation sequencing: discovery to translation

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Knocking down disease: a progress report on siRNA therapeutics

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Antisense technology: an emerging platform for cardiovascular disease therapeutics

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

The delivery of therapeutic oligonucleotides

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Gene-based therapies in lipidology: current status and future challenges

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

From mutation identification to therapy: discovery and origins of the first approved gene therapy in the Western world

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiency.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Lipodystrophy: metabolic insights from a rare disorder

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Phosphoinositide 3-kinase δ gene mutation predisposes to respiratory infection and airway damage

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Genetics. Simple genetics for a complex disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

The NHGRI GWAS Catalog, a curated resource of SNP-trait associations

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Schizophrenia risk from complex variation of complement component 4

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Advancing the understanding of autism disease mechanisms through genetics

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Validating therapeutic targets through human genetics

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Accelerating Medicines Partnership: Alzheimer's Disease (AMP-AD) Knowledge Portal Aids Alzheimer's Drug Discovery through Open Data Sharing

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

The genetics of drug efficacy: opportunities and challenges

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

The Path to Personalized Medicine

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Glivec (STI571, imatinib), a rationally developed, targeted anticancer drug

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Lessons from the cancer genome

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Emerging Agents and New Mutations in EGFR-Mutant Lung Cancer

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Reversible and adaptive resistance to BRAF(V600E) inhibition in melanoma

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Cocktails for cancer with a measure of immunotherapy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

DNA-Repair Defects and Olaparib in Metastatic Prostate Cancer

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Wild-type KRAS is required for panitumumab efficacy in patients with metastatic colorectal cancer

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Enhancement of antitumor immunity by CTLA-4 blockade

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

On being less tolerant: enhanced cancer immunosurveillance enabled by targeting checkpoints and agonists of T cell activation

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

The future of immune checkpoint therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Genetic basis for clinical response to CTLA-4 blockade in melanoma

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Cancer immunology. Mutational landscape determines sensitivity to PD-1 blockade in non-small cell lung cancer

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

CANCER. The odds of immunotherapy success

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Dissecting the multicellular ecosystem of metastatic melanoma by single-cell RNA-seq

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Adoptive cellular therapy: a race to the finish line.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Adoptive cell transfer as personalized immunotherapy for human cancer

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

New and emerging targeted therapies for cystic fibrosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Ataluren as an agent for therapeutic nonsense suppression

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Eteplirsen for the treatment of Duchenne muscular dystrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Mipomersen, an apolipoprotein B synthesis inhibitor, for lowering of LDL cholesterol concentrations in patients with homozygous familial hypercholesterolaemia: a randomised, double-blind, placebo-controlled trial

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Rapid progression of familial amyloidotic polyneuropathy: a multinational natural history study

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

An erythroid enhancer of BCL11A subject to genetic variation determines fetal hemoglobin level.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Functional footprinting of regulatory DNA.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Allele-Specific Inhibition of Rhodopsin With an Antisense Oligonucleotide Slows Photoreceptor Cell Degeneration

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Gene therapy returns to centre stage

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Gene therapy clinical trials worldwide to 2012 - an update.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

State of play in amyotrophic lateral sclerosis genetics

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Mechanisms of toxicity in C9FTLD/ALS.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Promising Targets for the Treatment of Neurodegenerative Diseases

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

C9orf72 nucleotide repeat structures initiate molecular cascades of disease.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Unconventional translation of C9ORF72 GGGGCC expansion generates insoluble polypeptides specific to c9FTD/ALS

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Poly-dipeptides encoded by the C9orf72 repeats bind nucleoli, impede RNA biogenesis, and kill cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

GGGGCC repeat expansion in C9orf72 compromises nucleocytoplasmic transport

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

The C9orf72 repeat expansion disrupts nucleocytoplasmic transport.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

C9orf72 BAC Transgenic Mice Display Typical Pathologic Features of ALS/FTD

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Human C9ORF72 Hexanucleotide Expansion Reproduces RNA Foci and Dipeptide Repeat Proteins but Not Neurodegeneration in BAC Transgenic Mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

C9orf72 is required for proper macrophage and microglial function in mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Clinical implications of recent breakthroughs in amyotrophic lateral sclerosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Antisense oligonucleotide therapy for neurodegenerative disease.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

RNA toxicity from the ALS/FTD C9ORF72 expansion is mitigated by antisense intervention

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Targeted degradation of sense and antisense C9orf72 RNA foci as therapy for ALS and frontotemporal degeneration

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Gain of Toxicity from ALS/FTD-Linked Repeat Expansions in C9ORF72 Is Alleviated by Antisense Oligonucleotides Targeting GGGGCC-Containing RNAs

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Exome sequencing in amyotrophic lateral sclerosis identifies risk genes and pathways

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Haploinsufficiency of TBK1 causes familial ALS and fronto-temporal dementia

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Quantitative proteomics reveal a feedforward mechanism for mitochondrial PARKIN translocation and ubiquitin chain synthesis.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Cell biology: Tagged tags engage disposal.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

The ubiquitin kinase PINK1 recruits autophagy receptors to induce mitophagy

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Optineurin is an autophagy receptor for damaged mitochondria in parkin-mediated mitophagy that is disrupted by an ALS-linked mutation

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Genomewide Association Studies and Common Disease — Realizing Clinical Utility

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Genetic variants associated with autoimmunity drive NFκB signaling and responses to inflammatory stimuli

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Genetics of Inflammatory Bowel Diseases

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Use of genome-wide association studies for drug repositioning

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Efficacy and safety of alirocumab in reducing lipids and cardiovascular events

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Efficacy and safety of evolocumab in reducing lipids and cardiovascular events.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Power of rare diseases: found in translation

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Building the foundation for genomics in precision medicine

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Individualized medicine from prewomb to tomb

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Epidemiology, epigenetics and the 'Gloomy Prospect': embracing randomness in population health research and practice.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Clinical development of an antisense therapy for the treatment of transthyretin-associated polyneuropathy

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

Romosozumab in Postmenopausal Women with Osteopenia

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW246

21 January 2018

Identifiers

DOI

10.1093/HMG/DDW246

0 references

1 reference