Related references
Note: Only part of the references are listed.FAP Neuropathy and Emerging Treatments
David Adams et al.
CURRENT NEUROLOGY AND NEUROSCIENCE REPORTS (2014)
Repurposing Diflunisal for Familial Amyloid Polyneuropathy A Randomized Clinical Trial
John L. Berk et al.
JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION (2013)
Safety and Efficacy of RNAi Therapy for Transthyretin Amyloidosis
Teresa Coelho et al.
NEW ENGLAND JOURNAL OF MEDICINE (2013)
Diagnosis and Therapeutic Approaches to Transthyretin Amyloidosis
Y. Ando et al.
CURRENT MEDICINAL CHEMISTRY (2012)
Tafamidis for transthyretin familial amyloid polyneuropathy A randomized, controlled trial
Teresa Coelho et al.
NEUROLOGY (2012)
Suppression of choroid plexus transthyretin levels by antisense oligonucleotide treatment
Merrill D. Benson et al.
AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS (2010)
The MIQE Guidelines: Minimum Information for Publication of Quantitative Real-Time PCR Experiments
Stephen A. Bustin et al.
CLINICAL CHEMISTRY (2009)
Enhancing transduction of the liver by adeno-associated viral vectors
A. C. Nathwani et al.
GENE THERAPY (2009)
Enhanced transthyretin tetramer stability following expression of an amyloid disease transsuppressor variant in mammalian cells
Masakazu Kamata et al.
JOURNAL OF GENE MEDICINE (2009)
Quantitative evaluation of DNA methylation by optimization of a differential-high resolution melt analysis protocol
Francesca Malentacchi et al.
NUCLEIC ACIDS RESEARCH (2009)
Gene Therapy Using Adeno-Associated Virus Vectors
Shyam Daya et al.
CLINICAL MICROBIOLOGY REVIEWS (2008)
Extracellular matrix markers for disease progression and follow-up of therapies in Familial Amyloid Polyneuropathy V30M TTR-related
I. Cardoso et al.
DISEASE MARKERS (2008)
Comparative in vitro and ex vivo activities of selected inhibitors of transthyretin aggregation:: relevance in drug design
Isabel Cardoso et al.
BIOCHEMICAL JOURNAL (2007)
Biomarkers in the assessment of therapies for familial amyloidotic polyneuropathy
Barbara Macedo et al.
MOLECULAR MEDICINE (2007)
High-resolution DNA melting analysis for simple and efficient molecular diagnostics
Gudrun H. Reed et al.
PHARMACOGENOMICS (2007)
Complete correction of enzymatic deficiency and neurochemistry in the GM1-gangliosidosis mouse brain by neonatal adeno-associated virus-mediated gene delivery
M. L. D. Broekman et al.
MOLECULAR THERAPY (2007)
Endoplasmic reticulum stress associated with extracellular aggregates - Evidence from transthyretin deposition in familial amyloid polyneuropathy
Pedro Filipe Teixeira et al.
JOURNAL OF BIOLOGICAL CHEMISTRY (2006)
Treatment of human disease by adeno-associated viral gene transfer
Kenneth H. Warrington et al.
HUMAN GENETICS (2006)
Targeted suppression of an amyloidogenic transthyretin with antisense oligonucleotides
MD Benson et al.
MUSCLE & NERVE (2006)
Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver
AC Nathwani et al.
BLOOD (2006)
Activation of ERK1/2 MAP kinases in familial amyloidotic polyneuropathy
FA Monteiro et al.
JOURNAL OF NEUROCHEMISTRY (2006)
Adeno-associated virus vectors serotyped with AAV8 capsid are more efficient than AAV-1 or-2 serotypes for widespread gene delivery to the neonatal mouse brain
MLD Broekman et al.
NEUROSCIENCE (2006)
Comparison of the ability of adeno-associated viral vectors pseudotyped with serotype 2, 5, and 8 capsid proteins to mediate efficient transduction of the liver in murine and nonhuman primate models
AM Davidoff et al.
MOLECULAR THERAPY (2005)
The biological and chemical basis for tissue-selective amyloid disease
Y Sekijima et al.
CELL (2005)
Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart
Z Wang et al.
NATURE BIOTECHNOLOGY (2005)
Unrestricted hepatocyte transduction with adeno-associated virus serotype 8 vectors in mice
H Nakai et al.
JOURNAL OF VIROLOGY (2005)
Selective binding to transthyretin and tetramer stabilization in serum from patients with familial amyloidotic polyneuropathy by an iodinated diflunisal derivative
MR Almeida et al.
BIOCHEMICAL JOURNAL (2004)
Sensitivity and specificity of single-nucleotide polymorphism scanning by high-resolution melting analysis
GH Reed et al.
CLINICAL CHEMISTRY (2004)
Targeted conversion of the transthyretin gene in vitro and in vivo
M Nakamura et al.
GENE THERAPY (2004)
Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer
F Mingozzi et al.
JOURNAL OF CLINICAL INVESTIGATION (2003)
Prevention of transthyretin amyloid disease by changing protein misfolding energetics
P Hammarström et al.
SCIENCE (2003)
Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo
DM McCarty et al.
GENE THERAPY (2003)
Trans-suppression of misfolding in an amyloid disease
P Hammarström et al.
SCIENCE (2001)
Tetramer dissociation and monomer partial unfolding precedes protofibril formation in amyloidogenic transthyretin variants
A Quintas et al.
JOURNAL OF BIOLOGICAL CHEMISTRY (2001)
Transthyretin stability as a key factor in amyloidogenesis: X-ray analysis at atomic resolution
MP Sebastiao et al.
JOURNAL OF MOLECULAR BIOLOGY (2001)
Share Paper
