Targeting protein aggregation for the treatment of degenerative diseases

YS Eisele, C Monteiro, C Fearns… - Nature reviews Drug …, 2015 - nature.com
The aggregation of specific proteins is hypothesized to underlie several degenerative
diseases, which are collectively known as amyloid disorders. However, the mechanistic …

Sickle cell disease

FB Piel, MH Steinberg, DC Rees - New England Journal of …, 2017 - Mass Medical Soc
Sickle cell disease is caused by an alteration in a single DNA base, but its clinical
manifestations are influenced by other genes and behavioral and environmental factors …

[HTML][HTML] Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease

EB Esrick, LE Lehmann, A Biffi, M Achebe… - … England Journal of …, 2021 - Mass Medical Soc
Background Sickle cell disease is characterized by hemolytic anemia, pain, and progressive
organ damage. A high level of erythrocyte fetal hemoglobin (HbF) comprising α-and γ …

The carboxy terminus of the. beta. amyloid protein is critical for the seeding of amyloid formation: Implications for the pathogenesis of Alzheimer's disease

JT Jarrett, EP Berger, PT Lansbury Jr - Biochemistry, 1993 - ACS Publications
MATERIALS AND METHODS Synthesis and Purification of Peptides. The peptides 026-39,
026-40, 026-42, and 026-43 were synthesized on the PAM resin using standard …

[PDF][PDF] Seeding “one-dimensional crystallization” of amyloid: a pathogenic mechanism in Alzheimer's disease and scrapie?

JT Jarrett, PT Lansbury Jr - Cell, 1993 - academia.edu
Alzheimer's disease (AD) is a neurodegenerative disease characterized by the presence of
cerebral amyloid plaque (reviewed by Selkoe, 1991), a highly ordered protein aggregate …

Treating sickle cell disease by targeting HbS polymerization

WA Eaton, HF Bunn - Blood, The Journal of the American …, 2017 - ashpublications.org
Although the root cause of sickle cell disease is the polymerization of hemoglobin S (HbS) to
form fibers that make red cells less flexible, most drugs currently being assessed in clinical …

Pathogenesis and treatment of sickle cell disease

HF Bunn - New England Journal of Medicine, 1997 - Mass Medical Soc
In 1949, the discovery that sickle hemoglobin (α2 βS2) has an abnormal electrophoretic
mobility prompted Linus Pauling and his colleagues to christen sickle cell anemia “a …

From macroscopic measurements to microscopic mechanisms of protein aggregation

SIA Cohen, M Vendruscolo, CM Dobson… - Journal of molecular …, 2012 - Elsevier
The ability to relate bulk experimental measurements of amyloid formation to the
microscopic assembly processes that underlie protein aggregation is critical in order to …

Protein aggregation kinetics, mechanism, and curve-fitting: a review of the literature

AM Morris, MA Watzky, RG Finke - … et Biophysica Acta (BBA)-Proteins and …, 2009 - Elsevier
Protein aggregation is an important phenomenon that alternatively is part of the normal
functioning of nature or, central to this review, has negative consequences via its …

Simulation studies of amyloidogenic polypeptides and their aggregates

IM Ilie, A Caflisch - Chemical reviews, 2019 - ACS Publications
Amyloids, fibrillar assembly of (poly) peptide chains, are associated with neurodegenerative
illnesses such as Alzheimer's and Parkinson's diseases, for which there are no cures. The …