Factors affecting the physical stability (aggregation) of peptide therapeutics
KL Zapadka, FJ Becher… - Interface …, 2017 - royalsocietypublishing.org
The number of biological therapeutic agents in the clinic and development pipeline has
increased dramatically over the last decade and the number will undoubtedly continue to …
increased dramatically over the last decade and the number will undoubtedly continue to …
Transthyretin (ATTR) amyloidosis: clinical spectrum, molecular pathogenesis and disease-modifying treatments
Y Sekijima - Journal of Neurology, Neurosurgery & Psychiatry, 2015 - jnnp.bmj.com
Transthyretin (ATTR) amyloidosis is a life-threatening, gain-of-toxic-function disease
characterised by extracellular deposition of amyloid fibrils composed of transthyretin (TTR) …
characterised by extracellular deposition of amyloid fibrils composed of transthyretin (TTR) …
Nonbiopsy diagnosis of cardiac transthyretin amyloidosis
Background—Cardiac transthyretin (ATTR) amyloidosis is a progressive and fatal
cardiomyopathy for which several promising therapies are in development. The diagnosis is …
cardiomyopathy for which several promising therapies are in development. The diagnosis is …
Safety and efficacy of RNAi therapy for transthyretin amyloidosis
Background Transthyretin amyloidosis is caused by the deposition of hepatocyte-derived
transthyretin amyloid in peripheral nerves and the heart. A therapeutic approach mediated …
transthyretin amyloid in peripheral nerves and the heart. A therapeutic approach mediated …
Tafamidis for transthyretin familial amyloid polyneuropathy: a randomized, controlled trial
T Coelho, LF Maia, A Martins da Silva… - Neurology, 2012 - AAN Enterprises
Objectives: To evaluate the efficacy and safety of 18 months of tafamidis treatment in
patients with early-stage V30M transthyretin familial amyloid polyneuropathy (TTR-FAP) …
patients with early-stage V30M transthyretin familial amyloid polyneuropathy (TTR-FAP) …
Rational design of cationic lipids for siRNA delivery
SC Semple, A Akinc, J Chen, AP Sandhu, BL Mui… - Nature …, 2010 - nature.com
We adopted a rational approach to design cationic lipids for use in formulations to deliver
small interfering RNA (siRNA). Starting with the ionizable cationic lipid 1, 2-dilinoleyloxy-3 …
small interfering RNA (siRNA). Starting with the ionizable cationic lipid 1, 2-dilinoleyloxy-3 …
[HTML][HTML] Stress-independent activation of XBP1s and/or ATF6 reveals three functionally diverse ER proteostasis environments
MD Shoulders, LM Ryno, JC Genereux, JJ Moresco… - Cell reports, 2013 - cell.com
The unfolded protein response (UPR) maintains endoplasmic reticulum (ER) proteostasis
through the activation of transcription factors such as XBP1s and ATF6. The functional …
through the activation of transcription factors such as XBP1s and ATF6. The functional …
Familial amyloid polyneuropathy
V Planté-Bordeneuve, G Said - The Lancet Neurology, 2011 - thelancet.com
Familial amyloid polyneuropathies (FAPs) are a group of life-threatening multisystem
disorders transmitted as an autosomal dominant trait. Nerve lesions are induced by deposits …
disorders transmitted as an autosomal dominant trait. Nerve lesions are induced by deposits …
Lipid-like materials for low-dose, in vivo gene silencing
KT Love, KP Mahon, CG Levins… - Proceedings of the …, 2010 - National Acad Sciences
Significant effort has been applied to discover and develop vehicles which can guide small
interfering RNAs (siRNA) through the many barriers guarding the interior of target cells …
interfering RNAs (siRNA) through the many barriers guarding the interior of target cells …
Protein aggregation diseases: pathogenicity and therapeutic perspectives
A Aguzzi, T O'connor - Nature reviews Drug discovery, 2010 - nature.com
A growing number of diseases seem to be associated with inappropriate deposition of
protein aggregates. Some of these diseases—such as Alzheimer's disease and systemic …
protein aggregates. Some of these diseases—such as Alzheimer's disease and systemic …