Results 121 to 130 of about 60,909 (346)

Engineered RNA Devices for In Vivo Targeted Therapeutics via Advanced Delivery Systems

open access: yesAggregate, EarlyView.
Schematic illustration of engineered RNA devices for in vivo targeted therapeutics via advanced delivery systems. ABSTRACT Engineered RNA devices can identify disease‐specific markers and precisely regulate gene expression, which is of great significance to the development of precision medicine.
Wei Luo   +6 more
wiley   +1 more source

Prevalence and characteristics of transthyretin amyloid cardiomyopathy in hypertrophic cardiomyopathy

open access: yesESC Heart Failure
Aims Recognition of transthyretin amyloid cardiomyopathy is increasing due to advances in cardiac imaging and diagnostic strategies, but questions remain regarding disease frequency and characteristics.
Pablo Garcia‐Pavia   +10 more
doaj   +1 more source

Synthesis and structural analysis of the N-terminal domain of the thyroid hormone-binding protein transthyretin [PDF]

open access: yes, 2002
Transthyretin (TTR) is a 55 kDa protein responsible for the transport of thyroid hormones and retinol in human serum. Misfolded forms of the protein are implicated in the amyloid diseases familial amyloidotic polyneuropathy and senile systemic ...
Hamilton JA   +9 more
core   +1 more source

Co‐Opting MBNL‐Dependent Alternative Splicing Cassette Exons to Control Gene Therapy in Myotonic Dystrophy

open access: yesAnnals of Neurology, EarlyView.
Objective Myotonic dystrophy type 1 (DM1) is a highly variable, multisystemic genetic disorder caused by a CTG repeat expansion in the 3′ untranslated region of DMPK. Toxicity is exerted by repeat‐containing DMPK transcripts that sequester muscleblind‐like (MBNL) proteins and lead to deleterious yet predictable changes in alternative splicing.
Samuel T. Carrell   +3 more
wiley   +1 more source

Serum Transthyretin Level as a Plausible Marker for Diagnosis of Child Acute Malnutrition

open access: yesBiochemistry Research International, 2017
Malnutrition is a major underlying condition for mortality in children under five years of age in developing countries, particularly in Ethiopia. The most important forms of malnutrition in Ethiopia are protein and energy deficiencies.
Behailu Tsegaye   +2 more
doaj   +1 more source

Creatinine, diet, micronutrients, and arsenic methylation in West Bengal, India. [PDF]

open access: yes, 2011
BackgroundIngested inorganic arsenic (InAs) is methylated to monomethylated (MMA) and dimethylated metabolites (DMA). Methylation may have an important role in arsenic toxicity, because the monomethylated trivalent metabolite [MMA(III)] is highly toxic ...
Basu, Arin   +9 more
core   +2 more sources

Intracellular Catalytic Photooxygenation of α‐Synuclein Amyloid

open access: yesChemistryEurope, EarlyView.
Amyloid‐selective catalytic photooxygenation reduces amyloid toxicity, aggregation tendencies, and seeding activities. Achieving therapeutic catalysis for intracellular amyloids is challenging due to conflicting oxygenation and reductive cellular environments.
Atsushi Iwai   +11 more
wiley   +1 more source

Familial amyloidosis with polyneuropathy type 1 caused by transthyretin mutation Val50Met (Val30Met): 4 cases in a non-endemic area [PDF]

open access: yes, 2018
Introduction: Transthyretin-related familial amyloid polyneuropathy (TTR-FAP) typically arises as an autonomic neuropathy primarily affecting small fibres and it occurs in adult patients in their second or third decades of life. It progresses rapidly and
Andrés, N.   +2 more
core   +2 more sources

Evolving Real‐World Data and Evidence Use for New Drugs and Regenerative Medical Products Approvals in Japan—An Analysis of the 6‐Year Trend

open access: yesClinical Pharmacology &Therapeutics, EarlyView.
Several regulatory initiatives have been made to clarify the acceptability and requirements of real‐world data and real‐world evidence (RWD/E) for the benefit/risk assessment of new medical products in Japan. The objectives of this review were to characterize the use of RWD/E in regulatory applications of new medical products and to describe the ...
Suguru Okami   +2 more
wiley   +1 more source

Low transthyretin concentration linked to adverse prognosis in elderly inpatients

open access: yesBMC Geriatrics
Background To investigate the association between low transthyretin (prealbumin) concentration and mortality or readmission for all causes in elderly inpatients.
Ting Wang   +9 more
doaj   +1 more source

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