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CRISPR-Cas9 in vivo gene editing for transthyretin amyloidosis

https://doi.org/10.1530/ey.19.14.13
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What this work says

Outgoing claims: the papers this work cites, builds on, or argues with.

inferredcites →https://doi.org/10.1002/ejhf.1695openalex-snapshot
Emerging Therapies in Transthyretin Amyloidosis – A New Wave of Hope After Years of Stagnancy?
inferredcites →https://doi.org/10.1002/mus.26034openalex-snapshot
Estimating the global prevalence of transthyretin familial amyloid polyneuropathy
inferredcites →https://doi.org/10.1006/gcen.2000.7520openalex-snapshot
Evolution of the Thyroid Hormone-Binding Protein, Transthyretin
inferredcites →https://doi.org/10.1007/s40120-020-00180-wopenalex-snapshot
Evaluation of Mortality During Long-Term Treatment with Tafamidis for Transthyretin Amyloidosis with Polyneuropathy: Clinical Trial Results up to 8.5 Years
inferredcites →https://doi.org/10.1016/j.cell.2014.05.010openalex-snapshot
Development and Applications of CRISPR-Cas9 for Genome Engineering
inferredcites →https://doi.org/10.1016/j.celrep.2018.02.014openalex-snapshot
A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editing
inferredcites →https://doi.org/10.1016/j.dnarep.2005.05.004openalex-snapshot
Chromosomal aberrations induced by double strand DNA breaks
inferredcites →https://doi.org/10.1016/j.jacc.2015.09.075openalex-snapshot
Diagnosis, Prognosis, and Therapy of Transthyretin Amyloidosis
inferredcites →https://doi.org/10.1016/j.ymthe.2018.03.010openalex-snapshot
A Novel Amino Lipid Series for mRNA Delivery: Improved Endosomal Escape and Sustained Pharmacology and Safety in Non-human Primates
inferredcites →https://doi.org/10.1016/s1474-4422(20)30368-9openalex-snapshot
Long-term safety and efficacy of patisiran for hereditary transthyretin-mediated amyloidosis with polyneuropathy: 12-month results of an open-label extension study
inferredcites →https://doi.org/10.1038/nbt.3117openalex-snapshot
GUIDE-seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases
inferredcites →https://doi.org/10.1038/nmeth.4284openalex-snapshot
Mapping the genomic landscape of CRISPR–Cas9 cleavage
inferredcites →https://doi.org/10.1038/s41392-019-0089-yopenalex-snapshot
Applications of genome editing technology in the targeted therapy of human diseases: mechanisms, advances and prospects
inferredcites →https://doi.org/10.1038/s41565-019-0591-yopenalex-snapshot
The Onpattro story and the clinical translation of nanomedicines containing nucleic acid-based drugs
inferredcites →https://doi.org/10.1038/s41588-020-0623-4openalex-snapshot
Cas9 activates the p53 pathway and selects for p53-inactivating mutations
inferredcites →https://doi.org/10.1038/s41591-018-0049-zopenalex-snapshot
CRISPR–Cas9 genome editing induces a p53-mediated DNA damage response
inferredcites →https://doi.org/10.1056/nejmoa1716153openalex-snapshot
Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis
inferredcites →https://doi.org/10.1056/nejmoa1805689openalex-snapshot
Tafamidis Treatment for Patients with Transthyretin Amyloid Cardiomyopathy
inferredcites →https://doi.org/10.1073/pnas.2135498100openalex-snapshot
Endogenous DNA double-strand breaks: Production, fidelity of repair, and induction of cancer
inferredcites →https://doi.org/10.1074/jbc.m008091200openalex-snapshot
Biochemical Basis for Depressed Serum Retinol Levels in Transthyretin-deficient Mice
inferredcites →https://doi.org/10.1093/bioinformatics/btu048openalex-snapshot
Cas-OFFinder: a fast and versatile algorithm that searches for potential off-target sites of Cas9 RNA-guided endonucleases
inferredcites →https://doi.org/10.1093/nar/gku402openalex-snapshot
CRISPR/Cas9 systems have off-target activity with insertions or deletions between target DNA and guide RNA sequences
inferredcites →https://doi.org/10.1111/ene.12225openalex-snapshot
Effect on disability and safety of Tafamidis in late onset of Met30 transthyretin familial amyloid polyneuropathy
inferredcites →https://doi.org/10.1111/jnc.15233openalex-snapshot
Targeting transthyretin ‐ Mechanism‐based treatment approaches and future perspectives in hereditary amyloidosis
inferredcites →https://doi.org/10.1126/science.1225829openalex-snapshot
A Programmable Dual-RNA–Guided DNA Endonuclease in Adaptive Bacterial Immunity
inferredcites →https://doi.org/10.1161/circheartfailure.119.006075openalex-snapshot
Expert Consensus Recommendations for the Suspicion and Diagnosis of Transthyretin Cardiac Amyloidosis
inferredcites →https://doi.org/10.1161/circulationaha.118.035831openalex-snapshot
Effects of Patisiran, an RNA Interference Therapeutic, on Cardiac Parameters in Patients With Hereditary Transthyretin-Mediated Amyloidosis
inferredcites →https://doi.org/10.1186/1750-1172-8-31openalex-snapshot
Guideline of transthyretin-related hereditary amyloidosis for clinicians
inferredcites →https://doi.org/10.15252/emmm.201505357openalex-snapshot
A novel mechano‐enzymatic cleavage mechanism underlies transthyretin amyloidogenesis
inferredcites →https://doi.org/10.3109/07853890.2015.1068949openalex-snapshot
Evolving landscape in the management of transthyretin amyloidosis
inferredcites →https://doi.org/10.4274/tnd.2019.57224openalex-snapshot
Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis

What points at this work

Incoming claims: where this work appears in other papers’ reference lists and arguments.

inferred← citeshttps://doi.org/10.2139/ssrn.4657914openalex-extracted
Delivery of Rna Drugs: Beyond the Liver, More than Vaccine
inferred← citeshttps://doi.org/10.1002/mco2.639openalex-snapshot
Gene editing therapy for cardiovascular diseases
inferred← citeshttps://doi.org/10.1002/psp4.13078openalex-snapshot
WITHDRAWAL: Quantitative modeling of approved and emerging therapeutics for modifying TTR levels in patients with Transthyretin Amyloid Cardiomyopathy
inferred← citeshttps://doi.org/10.1007/s00109-022-02199-yopenalex-snapshot
Current and future direction in treatment of HPV-related cervical disease
inferred← citeshttps://doi.org/10.1016/j.apmt.2021.101217openalex-snapshot
Multifunctional polymeric micelle-based nucleic acid delivery: Current advances and future perspectives
inferred← citeshttps://doi.org/10.1016/j.phrs.2023.106857openalex-snapshot
Recent advances in the management and implementation of care for familial hypercholesterolaemia
inferred← citeshttps://doi.org/10.1038/s41467-022-28028-xopenalex-snapshot
Systematic decomposition of sequence determinants governing CRISPR/Cas9 specificity
inferred← citeshttps://doi.org/10.1038/s41467-022-28244-5openalex-snapshot
CRISPR-Cas9 induces large structural variants at on-target and off-target sites in vivo that segregate across generations
inferred← citeshttps://doi.org/10.1063/5.0115407openalex-snapshot
Applications and challenges for CRISPR/Cas9-mediated gene editing
inferred← citeshttps://doi.org/10.1080/13506129.2021.2018678openalex-snapshot
The integration of genetically-regulated transcriptomics and electronic health records highlights a pattern of medical outcomes related to increased hepatic transthyretin expression
inferred← citeshttps://doi.org/10.1093/med/9780197549469.003.0059openalex-snapshot
What Rodent Models Teach Us about the Association of Autism and Epilepsy
inferred← citeshttps://doi.org/10.1093/procel/pwac052openalex-snapshot
An engineered xCas12i with high activity, high specificity and broad PAM range
inferred← citeshttps://doi.org/10.1101/2024.04.02.586657openalex-snapshot
Cas9 RNP Physiochemical Analysis for Enhanced CRISPR-AuNP Assembly and Function
inferred← citeshttps://doi.org/10.1101/2024.04.16.587991openalex-snapshot
Repair of leukemia-associated single nucleotide variants via interallelic gene conversion
inferred← citeshttps://doi.org/10.1158/2159-8290.c.6602824openalex-snapshot
Data from A View on Drug Development for Cancer Prevention
inferred← citeshttps://doi.org/10.1158/2159-8290.c.6602824.v1openalex-snapshot
Data from A View on Drug Development for Cancer Prevention
inferred← citeshttps://doi.org/10.18203/2320-6012.ijrms20241589openalex-snapshot
CRISPR/Cas9-mediated genome editing: from basic research to gene therapy
inferred← citeshttps://doi.org/10.21203/rs.3.rs-1743660/v1openalex-snapshot
Genetic deletion of HPV E7 oncogene effectively eliminates HPV driven oropharyngeal cancer tumours
inferred← citeshttps://doi.org/10.21203/rs.3.rs-1836086/v1openalex-snapshot
Early Detection and Disease Monitoring of Hepatocellular Carcinoma Using Circulating Telomere DNA
inferred← citeshttps://doi.org/10.21203/rs.3.rs-2386865/v1openalex-snapshot
A Peptide Barcode Based Approach for Efficient in vivo Screening of RNA Delivery Systems
inferred← citeshttps://doi.org/10.22541/au.165268627.74823408/v1openalex-snapshot
The Control and Trust of CRISPR / Cas9 Genome Altering for Clinical Application with Gene Treatment and Treatment of Hereditary Diseases
inferred← citeshttps://doi.org/10.22541/au.165493454.42208986/v1openalex-snapshot
New frontiers in CRISPR/Cas9 delivery systems delivery for gene editing
inferred← citeshttps://doi.org/10.32689/2663-0672-2023-3-2openalex-snapshot
GENOME EDITING: CLUSTERED REGULARLY INTERSPACED SHORT PALINDROMIC REPEAT (CRISPR/CAS9)
inferred← citeshttps://doi.org/10.32756/0869-5490-2024-2openalex-snapshot
inferred← citeshttps://doi.org/10.33540/1102openalex-snapshot
The road towards precision therapy for genetic epilepsies
inferred← citeshttps://doi.org/10.33540/2032openalex-snapshot
Lipid Nanoparticle-Mediated Delivery of CRISPR-Cas9 for Therapeutic Gene Correction
inferred← citeshttps://doi.org/10.3389/fonc.2026.1888734openalex-snapshot
CRISPR/Cas9 in cancer therapy: clinical translation, mechanistic strategies, and therapeutic directions
inferred← citeshttps://doi.org/10.3390/molecules30030542openalex-snapshot
CRISPR-Cas9 Gene Therapy: Non-Viral Delivery and Stimuli-Responsive Nanoformulations
inferred← citeshttps://doi.org/10.3390/ph14080765openalex-snapshot
Chromatin Alterations in Neurological Disorders and Strategies of (Epi)Genome Rescue
inferred← citeshttps://doi.org/10.3390/v13071373openalex-snapshot
Immunity and Viral Infections: Modulating Antiviral Response via CRISPR–Cas Systems
inferred← citeshttps://doi.org/10.37868/bes.v3i1.id215openalex-snapshot
CRISPR-Cas9: A new approach to genome editing and clinical treatment
inferred← citeshttps://doi.org/10.3791/64558openalex-snapshot
Engineering Oncogenic Heterozygous Gain-of-Function Mutations in Human Hematopoietic Stem and Progenitor Cells
inferred← citeshttps://doi.org/10.3791/64558-vopenalex-snapshot
Engineering Oncogenic Heterozygous Gain-of-Function Mutations in Human Hematopoietic Stem and Progenitor Cells
inferred← citeshttps://doi.org/10.4337/9781035302055.00007openalex-snapshot
Introduction: regulation of biotechnology, between anxieties and trust in scientific progress
inferred← citeshttps://doi.org/10.52102/diabet/pract/2022.1/art2openalex-snapshot
Diabetes, genética y epigenética: cómo aplicar la medicina personalizada de precisión
inferred← citeshttps://doi.org/10.53730/ijhs.v6ns10.15029openalex-snapshot
Advanced drug delivery systems for enhancing the efficacy of RNA-based therapeutics
inferred← citeshttps://doi.org/10.53846/goediss-10501openalex-snapshot
Functional CRISPR repair of induced pluripotent stem cells from patients with Noonan syndrome-associated cardiac hypertrophy
inferred← citeshttps://doi.org/10.70675/e986610fz5953z4cb5z90b1zb23e0c1b1125openalex-snapshot
Épidémiologie génétique et estimation du risque de maladie (pénétrance) des amyloses héréditaires à transthyrétine
inferred← citeshttps://doi.org/10.7150/thno.84291openalex-snapshot
Nanoparticle technology for mRNA: Delivery strategy, clinical application and developmental landscape
inferred← citeshttps://doi.org/10.7150/thno.88550openalex-snapshot
Targeting DAD1 gene with CRISPR-Cas9 system transmucosally delivered by fluorinated polylysine nanoparticles for bladder cancer intravesical gene therapy
inferred← citeshttps://doi.org/10.7150/thno.90071openalex-snapshot
Quaternization drives spleen-to-lung tropism conversion for mRNA-loaded lipid-like nanoassemblies
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