Delivery of oligonucleotide‐based therapeutics: challenges and opportunities

SM Hammond, A Aartsma‐Rus, S Alves… - EMBO molecular …, 2021 - embopress.org
Nucleic acid‐based therapeutics that regulate gene expression have been developed
towards clinical use at a steady pace for several decades, but in recent years the field has …

Imaging of amyloid β in Alzheimer's disease with 18F-BAY94-9172, a novel PET tracer: proof of mechanism

CC Rowe, U Ackerman, W Browne, R Mulligan… - The Lancet …, 2008 - thelancet.com
Background Amyloid-β (Aβ) plaque formation is a hallmark of Alzheimer's disease (AD) and
precedes the onset of dementia. Aβ imaging should allow earlier diagnosis, but clinical …

Cloning of cDNAs encoding mammalian double-stranded RNA-specific adenosine deaminase

MA O'Connell, S Krause, M Higuchi… - … and cellular biology, 1995 - Taylor & Francis
Double-stranded RNA (dsRNA)-specific adenosine deaminase converts adenosine to
inosine in dsRNA. The protein has been purified from calf thymus, and here we describe the …

Ultrastructural analysis of transcription and splicing in the cell nucleus after bromo-UTP microinjection

D Cmarko, PJ Verschure, TE Martin… - Molecular biology of …, 1999 - Am Soc Cell Biol
In this study we demonstrate, at an ultrastructural level, the in situ distribution of
heterogeneous nuclear RNA transcription sites after microinjection of 5-bromo-UTP (BrUTP) …

A founder mutation in Anoctamin 5 is a major cause of limb girdle muscular dystrophy

D Hicks, A Sarkozy, N Muelas, K Köehler, A Huebner… - Brain, 2011 - academic.oup.com
The limb girdle muscular dystrophies (LGMDs) are a group of disorders with wide genetic
and clinical heterogeneity. Recently, mutations in the ANO5 gene, which encodes a putative …

Curcumin‐derived pyrazoles and isoxazoles: Swiss army knives or blunt tools for Alzheimer's disease?

R Narlawar, M Pickhardt… - ChemMedChem …, 2008 - Wiley Online Library
Curcumin binds to the amyloid β peptide (Aβ) and inhibits or modulates amyloid precursor
protein (APP) metabolism. Therefore, curcumin‐derived isoxazoles and pyrazoles were …

Dystrophin-deficient pigs provide new insights into the hierarchy of physiological derangements of dystrophic muscle

N Klymiuk, A Blutke, A Graf, S Krause… - Human molecular …, 2013 - academic.oup.com
Duchenne muscular dystrophy (DMD) is caused by mutations in the X-linked dystrophin
(DMD) gene. The absence of dystrophin protein leads to progressive muscle weakness and …

[PDF][PDF] Hexosamine biosynthetic pathway mutations cause neuromuscular transmission defect

J Senderek, JS Müller, M Dusl, TM Strom… - The American Journal of …, 2011 - cell.com
Neuromuscular junctions (NMJs) are synapses that transmit impulses from motor neurons to
skeletal muscle fibers leading to muscle contraction. Study of hereditary disorders of …

Pathological consequences of VCP mutations on human striated muscle

CU Hübbers, CS Clemen, K Kesper, A Böddrich… - Brain, 2007 - academic.oup.com
Mutations in the valosin-containing protein (VCP, p97) gene on chromosome 9p13–p12
cause a late-onset form of autosomal dominant inclusion body myopathy associated with …

TraG-Like Proteins of DNA Transfer Systems and of the Helicobacter pylori Type IV Secretion System: Inner Membrane Gate for Exported Substrates?

G Schröder, S Krause, EL Zechner… - Journal of …, 2002 - Am Soc Microbiol
TraG-like proteins are potential NTP hydrolases (NTPases) that are essential for DNA
transfer in bacterial conjugation. They are thought to mediate interactions between the DNA …