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Genetics & Rare Diseases

Why A Gene Therapy Pioneer Is Raising Concerns About Treatments He Championed

16 Feb, 2018 | 00:40h | UTC

Why A Gene Therapy Pioneer Is Raising Concerns About Treatments He Championed – Forbes (free)

 


Review: Congenital Insensitivity to Pain Overview

16 Feb, 2018 | 00:36h | UTC

Congenital Insensitivity to Pain Overview – GeneReviews (free)

 


The Famine Ended 70 Years Ago, but Dutch Genes Still Bear Scars

15 Feb, 2018 | 11:11h | UTC

The Famine Ended 70 Years Ago, but Dutch Genes Still Bear Scars – The New York Times (free)

 


Research: Polygenic Contribution in Individuals With Early-Onset Coronary Artery Disease

15 Feb, 2018 | 10:39h | UTC

Polygenic Contribution in Individuals With Early-Onset Coronary Artery Disease – Circulation: Genomic and Precision Medicine (free)

Commentary: Genetic Risk Score May Best FH Variant for Predicting Early-Onset CAD – Medscape (free registration required)

“Another study suggests CAD will soon be predicted not by 1 gene (like FH) but groups of genes that can be combined into risk scores” (via @drjohnm see Tweet)

 


Review: Management of Turner Syndrome

4 Feb, 2018 | 16:57h | UTC

Current best practice in the management of Turner syndrome – Therapeutic Advances in Endocrinology and Metabolism (free)

 


Research: Cloning of Macaque Monkeys

26 Jan, 2018 | 00:38h | UTC

Cloning of Macaque Monkeys by Somatic Cell Nuclear Transfer – Cell (free)

Commentaries: These monkey twins are the first primate clones made by the method that developed Dolly – Science (free) AND First monkeys cloned with technique that made Dolly the sheep – Nature (free) AND How afraid of human cloning should we be? – The Guardian (free)

 


Gene Editing – And What it Really Means to Rewrite the Code of Life

25 Jan, 2018 | 20:53h | UTC

Gene editing – and what it really means to rewrite the code of life – The Guardian (free)

Related: Correction of a pathogenic gene mutation in human embryos (free articles and commentaries) AND Simple guide to CRISPR, one of the biggest science stories of the decade – VOX (free)

 


Ethical and Safety Issues of Stem Cell-Based Therapy

23 Jan, 2018 | 14:18h | UTC

Ethical and Safety Issues of Stem Cell-Based Therapy – International Journal of Medical Science (free)

 


Supervised Machine Learning for Population Genetics

23 Jan, 2018 | 14:04h | UTC

Supervised Machine Learning for Population Genetics: A New Paradigm – Trends in Genetics (free)

 


Representing a “Revolution”: How the Popular Press has Portrayed Personalized Medicine

16 Jan, 2018 | 17:14h | UTC

Representing a “revolution”: how the popular press has portrayed personalized medicine – Genetics in Medicine (free)

 


Review of Genetic Reticulate Pigmentary Disorders

16 Jan, 2018 | 13:29h | UTC

Updated Review of Genetic Reticulate Pigmentary Disorders – The British Journal of Dermatology, via Medscape (free registration required)

 


Research: Germline BRCA Mutation and Outcome in Young-Onset Breast Cancer

16 Jan, 2018 | 12:57h | UTC

Germline BRCA mutation and outcome in young-onset breast cancer (POSH): a prospective cohort study – The Lancet Oncology (free)

Commentaries: Breast cancer in young women: do BRCA1 or BRCA2 mutations matter? – The Lancet Oncology (free) AND Survival for young breast cancer patients is the same whether or not they carry a BRCA mutation – eCancer News (free) AND Young-Onset Breast Cancer Survival Unaffected by BRCA Status – MedPage Today (free)

 


Review: Emerging Models of Direct-to-Consumer Genetic Testing

9 Jan, 2018 | 23:58h | UTC

Direct-to-Consumer Testing 2.0: Emerging Models of Direct-to-Consumer Genetic Testing – Mayo Clinic Proceedings (free)

 


$850,000 per Patient for Blindness Gene Therapy

4 Jan, 2018 | 16:53h | UTC

Spark to charge $850,000 per patient for blindness gene therapy – Reuters (free) AND US drug firm offers cure for blindness – at $425,000 an eye – The Guardian (free) AND Spark prices its gene therapy as most expensive U.S. medicine — but with plans to ease cost concerns – STAT (free)

 


Review: Medical Management of Aortic Disease in Marfan Syndrome

2 Jan, 2018 | 16:01h | UTC

Medical management of aortic disease in Marfan syndrome – Annals of Cardiothoracic Surgery (free)

 


CRISPR-ing Humans in 2018

29 Dec, 2017 | 17:49h | UTC

“CRISPR-ing humans in 2018: gearing up for many clinical trials X3” (RT @EricTopol see Tweet)

– CRISPR in 2018: Coming to a Human Near You – MIT Technology Review (free)

– In 2018, We Will CRISPR Human Beings – GIZMODO (free)

– Human CRISPR Trials Will Happen in 2018. They’ll Look Like This – BigThink (free)

Related: Simple guide to CRISPR, one of the biggest science stories of the decade – VOX (free)

 


Consensus: Role of Genetic Testing for Inherited Prostate Cancer Risk

27 Dec, 2017 | 21:19h | UTC

Role of Genetic Testing for Inherited Prostate Cancer Risk: Philadelphia Prostate Cancer Consensus Conference 2017 – Journal of Clinical Oncology (free)

Commentary: New Recommendations Guide Doctors on Genetic Counseling and Genetic Testing for Hereditary Prostate Cancer – Thomas Jefferson University, via NewsWise (free)

 


Review – SDHx mutations: beyond pheochromocytomas and paragangliomas

27 Dec, 2017 | 20:44h | UTC

SDHx mutations: beyond pheochromocytomas and paragangliomas – European Journal of Endocrinology (free)

 


Guideline: Genetic/Familial High-Risk Assessment in Colorectal Cancer

26 Dec, 2017 | 19:58h | UTC

NCCN Guidelines Insights: Genetic/Familial High-Risk Assessment: Colorectal, Version 3.2017 – Journal of the National Comprehensive Cancer Network (free)


Review of Prader-Willi Syndrome: The Endocrine Approach

26 Dec, 2017 | 19:27h | UTC

Review of Prader-Willi syndrome: the endocrine approach – Translational Pediatrics (free)

 


The best and worst analogies for CRISPR, ranked

12 Dec, 2017 | 12:07h | UTC

The best and worst analogies for CRISPR, ranked – STAT (free)

Related: Simple guide to CRISPR, one of the biggest science stories of the decade – VOX (free)

 


Research: Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant

7 Dec, 2017 | 22:06h | UTC

Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant – New England Journal of Medicine (link to abstract – $ for full-text)

Quick Take Video Summary: Gene Therapy for Factor IX Deficiency (free)

Commentary: One-dose gene therapy produces clotting factor, safely stops bleeding in hemophilia B patients – MedicalXpress (free)

 


Overview of the cutaneous porphyrias

5 Dec, 2017 | 18:13h | UTC

An overview of the cutaneous porphyrias – F1000 Research (free)

 


European guidelines on phenylketonuria

3 Dec, 2017 | 17:41h | UTC

The complete European guidelines on phenylketonuria: diagnosis and treatment – Orphanet Journal of Rare Diseases (free)

 


Review: Recent Advances in Understanding Werner Syndrome

3 Dec, 2017 | 17:15h | UTC

Recent Advances in Understanding Werner Syndrome – F1000 Research (free)

 


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