Results 41 to 50 of about 16,120 (219)

Left Bundle Branch Pacing in Transthyretin Cardiac Amyloidosis and Alternating Bundle Branch Block

JACC: Case Reports, 2020
Progressive conduction system disease affects patients with transthyretin cardiac amyloidosis, often requiring permanent pacing as the His-Purkinje system is affected. We present a case of left bundle branch pacing in a patient with transthyretin cardiac
Taha Ahmed, MD, Samra Haroon Lodhi, MBBS, Hassan Lak, MD, Jae Lee, MD, Mazen Hanna, MD, Roy Chung, MD   +5 more
doaj   +1 more source

Nonviral Gold Nanoparticle‐Mediated Delivery of CRISPR‐Cas9 Ribonucleoprotein and Long DNA Transgenes Into Primary Blood Cells

Advanced NanoBiomed Research, EarlyView.
CRISPR/Cas9 has revolutionized the field of gene therapy, but delivery remains an outstanding issue. We propose a nonviral gold‐nanoparticle platform for co‐delivery of CRISPR/Cas9 ribonucleoprotein and long 2.1 kilobase dsDNA transgene constructs. This CRISPR‐AuNP is inexpensive to produce and mediate gene editing and DNA delivery in T cells and CD34+
Rachel A. Cunningham, Karthikeya S. V. Gottimukkala, Daniel D. Lane, Katrina Poljakov, Patricia Lipson, Mark R. Enstrom, Alessandro Rizzi, Aude G. Chapuis, Jennifer E. Adair   +8 more
wiley   +1 more source

Misdiagnosis of hereditary amyloidosis as AL (Primary) amyloidosis [PDF]

, 2002
Background: Hereditary, autosomal dominant amyloidosis, caused by mutations in the genes encoding transthyretin, fibrinogen A -chain, lysozyme, or apolipoprotein A-I, is thought to be extremely rare and is not routinely included in the differential ...
Booth, D.R., Gilbertson, J.A., Hawkins, P.N., Booth, S.E., Lachmann, H.J., Gillmore, J.D., Bybee, A., Pepys, M.B.   +7 more
core  

Oligonucleotide Drugs for Transthyretin Amyloidosis [PDF]

New England Journal of Medicine, 2018
In this issue of the Journal, Adams et al.1 and Benson et al.2 report the results of two randomized, double-blind, controlled trials testing the therapeutic efficacy of two different chemically mod...
Adams, D, Hawkins, PN, Polydefkis, M
openaire   +6 more sources

Decoding RNA regulation: Challenges and opportunities for RNA‐based therapies in Europe

British Journal of Clinical Pharmacology, EarlyView.
Abstract RNA‐based medicinal products represent a promising frontier in personalised medicine, offering sequence‐specific disease targeting at various molecular levels, yet their clinical translation in the European Union (EU) may be hindered by regulatory uncertainty around definitions and evidence requirements; this study therefore aims to identify ...
Olivia C. Lewis, Christine Leopold, Joyce M. Hoek, Raymond M. Schiffelers, Marie L. De Bruin   +4 more
wiley   +1 more source

Quality of life assessment in amyloid transthyretin (ATTR) amyloidosis [PDF]

, 2021
Background: Amyloid transthyretin (ATTR) amyloidosis is caused by the systemic deposition of transthyretin molecules, either normal (wild-type ATTR, ATTRwt) or mutated (variant ATTR, ATTRv). ATTR amyloidosis is a disease with a severe impact on patients’
Cappelli F., Zampieri M., Milani P., Rapezzi C., Palladini G., Teresi L., Obici L., Perfetto F., Panichella G., De Rosis S., Licordari R., Quattrone F., Passino C., Ribarich N., Aimo A., Castiglione V., Emdin M., Merlini G., Di Bella G., Vergaro G., Serenelli M.   +20 more
core   +1 more source

Technetium-99m-labeled pyrophosphate uptake in the rectum of a patient with wild-type transthyretin cardiac amyloidosis

Radiology Case Reports
Wild-type transthyretin amyloidosis is a disease characterized by deposition of transthyretin amyloid protein in systemic organs and tissues, especially the heart, lungs, tenosynovium, and ligaments.
Koji Takahashi, MD, PhD, Daisuke Sasaki, MRT, Shuhei Yamamoto, MD, Nobuhisa Yamamura, MLT, Yushi Utsunomiya, MD, Hiroe Morioka, MD, Shigeki Uemura, MD, Tomoki Sakaue, MD, PhD, Katsuji Inoue, MD, PhD   +8 more
doaj   +1 more source

Cerebrovascular Events in Aortic Stenosis: From Native Valve Disease to TAVR‐Specific Risk and Prevention

Catheterization and Cardiovascular Interventions, EarlyView.
ABSTRACT Aortic stenosis (AS) is associated with a heightened burden of cardiovascular comorbidities, atrial fibrillation (AF), and progressive valvular calcification, all of which may contribute to cerebrovascular events across the disease continuum.
Priyanka Boettger, Jamschid Sedighi, Martin Juenemann, Omar Alhaj‐Omar, Kerstin Piayda, Mani Arsalan, Samuel Sossalla, Won‐Keun Kim   +7 more
wiley   +1 more source

Transthyretin Cardiac Amyloidosis: Pathogenesis, Treatments, and Emerging Role in Heart Failure with Preserved Ejection Fraction

, 2014
Transthyretin (TTR) amyloidosis causes heart failure from cardiac deposition of TTR amyloid fibrils, the by-product of TTR homotetramer disassembly. Wild-type (WT) TTR deposition leads to senile amyloidosis, predominantly manifesting with cardiomyopathy.
Daniel P. Judge, Van-Khue Ton, Monica Mukherjee   +2 more
core   +1 more source

Structure-Based Probe Reveals the Presence of Large Transthyretin Aggregates in Plasma of ATTR Amyloidosis Patients

JACC: Basic to Translational Science
Summary: Amyloidogenic transthyretin (ATTR) amyloidosis is a relentlessly progressive disease caused by the misfolding and systemic accumulation of amyloidogenic transthyretin into amyloid fibrils.
Rose Pedretti, BS, Lanie Wang, BS, Anna Yakubovska, MS, Qiongfang S. Zhang, BS, Binh Nguyen, PhD, Justin L. Grodin, MD, Ahmad Masri, MD, Lorena Saelices, PhD   +7 more
doaj   +1 more source