Julia F. Alterman
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View article: 189 STAR Particles: A Breakthrough Solution for long-lasting, safe, and effective siRNA skin Delivery
189 STAR Particles: A Breakthrough Solution for long-lasting, safe, and effective siRNA skin Delivery Open
View article: Systematic analysis of siRNA and mRNA features impacting fully chemically modified siRNA efficacy
Systematic analysis of siRNA and mRNA features impacting fully chemically modified siRNA efficacy Open
Chemically modified small interfering RNAs (siRNAs) are a promising drug class that silences disease-causing genes via mRNA degradation. Both siRNA-specific features (e.g. sequence, modification pattern, and structure) and target mRNA-spec…
View article: A systemically deliverable lipid-conjugated siRNA targeting DUX4 as an facioscapulohumeral muscular dystrophy therapeutic
A systemically deliverable lipid-conjugated siRNA targeting DUX4 as an facioscapulohumeral muscular dystrophy therapeutic Open
View article: Lowering the <i>HTT1a</i> transcript as an effective therapy for Huntington’s disease
Lowering the <i>HTT1a</i> transcript as an effective therapy for Huntington’s disease Open
Lowering the levels of HTT transcripts has been a major focus of therapeutic development for Huntington’s disease (HD), but which transcript should be lowered? HD is caused by a CAG repeat expansion in exon 1 of the HTT gene, and the rate …
View article: A Therapeutic Small-Interfering RNA Potentiates Janus Kinase 1 Modulation for the Treatment of Dog Inflammatory Diseases
A Therapeutic Small-Interfering RNA Potentiates Janus Kinase 1 Modulation for the Treatment of Dog Inflammatory Diseases Open
Inflammatory cytokine signaling pathways share notable similarities between humans and dogs. Janus kinase (JAK) family enzymesJAK1, JAK2, JAK3, and tyrosine kinase 2 (TYK2) are popular therapeutic targets for inflammatory diseases in hum…
View article: Divalent siRNA for prion disease
Divalent siRNA for prion disease Open
Pharmacologic lowering of PrP expression is efficacious against prion disease in animal models and is now being tested clinically. 50% lowering of PrP increases both survival time and healthy life in prion-infected mice, but does not preve…
View article: Preventing acute neurotoxicity of CNS therapeutic oligonucleotides with the addition of Ca2+ and Mg2+ in the formulation
Preventing acute neurotoxicity of CNS therapeutic oligonucleotides with the addition of Ca2+ and Mg2+ in the formulation Open
Oligonucleotide therapeutics (ASOs and siRNAs) have been explored for modulation of gene expression in the central nervous system (CNS), with several drugs approved and many in clinical evaluation. Administration of highly concentrated oli…
View article: Preventing acute neurotoxicity of CNS therapeutic oligonucleotides with the addition of Ca2+ and Mg2+ in the formulation
Preventing acute neurotoxicity of CNS therapeutic oligonucleotides with the addition of Ca2+ and Mg2+ in the formulation Open
Oligonucleotide therapeutics (ASOs and siRNAs) have been explored for modulation of gene expression in the central nervous system (CNS), with several drugs approved and many in clinical evaluation. Administration of highly concentrated oli…
View article: A programmable dual-targeting siRNA scaffold supports potent two-gene modulation in the central nervous system
A programmable dual-targeting siRNA scaffold supports potent two-gene modulation in the central nervous system Open
Divalent short-interfering RNA (siRNA) holds promise as a therapeutic approach allowing for the sequence-specific modulation of a target gene within the central nervous system (CNS). However, an siRNA modality capable of simultaneously mod…
View article: A combinatorial approach for achieving CNS-selective RNAi
A combinatorial approach for achieving CNS-selective RNAi Open
RNA interference (RNAi) is an endogenous process that can be harnessed using chemically modified small interfering RNAs (siRNAs) to potently modulate gene expression in many tissues. The route of administration and chemical architecture ar…
View article: Multispecies-targeting siRNAs for the modulation of JAK1 in the skin
Multispecies-targeting siRNAs for the modulation of JAK1 in the skin Open
View article: A programmable dual-targeting di-valent siRNA scaffold supports potent multi-gene modulation in the central nervous system
A programmable dual-targeting di-valent siRNA scaffold supports potent multi-gene modulation in the central nervous system Open
Di-valent short interfering RNA (siRNA) is a promising therapeutic modality that enables sequence-specific modulation of a single target gene in the central nervous system (CNS). To treat complex neurodegenerative disorders, where pathogen…
View article: Single intravitreal administration of a tetravalent siRNA exhibits robust and efficient gene silencing in mouse and pig photoreceptors
Single intravitreal administration of a tetravalent siRNA exhibits robust and efficient gene silencing in mouse and pig photoreceptors Open
View article: Rational design of a JAK1-selective siRNA inhibitor for the modulation of autoimmunity in the skin
Rational design of a JAK1-selective siRNA inhibitor for the modulation of autoimmunity in the skin Open
View article: Single intravitreal administration of a tetravalent siRNA exhibits robust and efficient gene silencing in rodent and swine photoreceptors
Single intravitreal administration of a tetravalent siRNA exhibits robust and efficient gene silencing in rodent and swine photoreceptors Open
Inherited retinal dystrophies caused by dominant mutations in photoreceptor-expressed genes, are a major cause of irreversible vision loss. Oligonucleotide therapy has been of interest in diseases that conventional medicine cannot target. …
View article: DIPG-17. TREATMENT OF DIFFUSE INTRINSIC PONTINE GLIOMA USING ALLELE-SPECIFIC SIRNA TARGETING H3.3 K27M MUTANT HISTONE
DIPG-17. TREATMENT OF DIFFUSE INTRINSIC PONTINE GLIOMA USING ALLELE-SPECIFIC SIRNA TARGETING H3.3 K27M MUTANT HISTONE Open
In recent years CNS tumors have surpassed leukemia and lymphoma as the deadliest forms of pediatric cancer, yet many of these tumors remain resistant to treatment. Understanding of the molecular pathology of a particularly fatal pediatric …
View article: Extended Nucleic Acid (exNA): A Novel, Biologically Compatible Backbone that Significantly Enhances Oligonucleotide Efficacy in vivo
Extended Nucleic Acid (exNA): A Novel, Biologically Compatible Backbone that Significantly Enhances Oligonucleotide Efficacy in vivo Open
View article: Extended Nucleic Acid (exNA): A Novel, Biologically Compatible Backbone that Significantly Enhances Oligonucleotide Efficacy<i>in vivo</i>
Extended Nucleic Acid (exNA): A Novel, Biologically Compatible Backbone that Significantly Enhances Oligonucleotide Efficacy<i>in vivo</i> Open
Metabolic stabilization of therapeutic oligonucleotides requires both sugar and backbone modifications, where phosphorothioate (PS) is the only backbone chemistry used in the clinic. Here, we describe the discovery, synthesis, and characte…
View article: Chemical engineering of therapeutic siRNAs for allele-specific gene silencing in Huntington’s disease models
Chemical engineering of therapeutic siRNAs for allele-specific gene silencing in Huntington’s disease models Open
Small interfering RNAs are a new class of drugs, exhibiting sequence-driven, potent, and sustained silencing of gene expression in vivo. We recently demonstrated that siRNA chemical architectures can be optimized to provide efficient deliv…
View article: Chemical engineering of therapeutic siRNAs for allele-specific gene silencing<i>in vivo</i>in CNS
Chemical engineering of therapeutic siRNAs for allele-specific gene silencing<i>in vivo</i>in CNS Open
Small interfering RNAs (siRNAs) are a new class of drugs, exhibiting sequence-driven, potent, and sustained silencing of gene expression in vivo . We recently demonstrated that siRNA chemical architectures can be optimized to provide effic…
View article: Chemical optimization of siRNA for safe and efficient silencing of placental sFLT1
Chemical optimization of siRNA for safe and efficient silencing of placental sFLT1 Open
Preeclampsia (PE) is a rising, potentially lethal complication of pregnancy. PE is driven primarily by the overexpression of placental soluble fms-like tyrosine kinase 1 (sFLT1), a validated diagnostic and prognostic marker of the disease …
View article: PK-modifying anchors significantly alter clearance kinetics, tissue distribution, and efficacy of therapeutics siRNAs
PK-modifying anchors significantly alter clearance kinetics, tissue distribution, and efficacy of therapeutics siRNAs Open
View article: Comparative route of administration studies using therapeutic siRNAs show widespread gene modulation in Dorset sheep
Comparative route of administration studies using therapeutic siRNAs show widespread gene modulation in Dorset sheep Open
siRNAs comprise a class of drugs that can be programmed to silence any target gene. Chemical engineering efforts resulted in development of divalent siRNAs (di-siRNAs), which support robust and long-term efficacy in rodent and nonhuman pri…
View article: An RNAi therapeutic targeting hepatic DGAT2 in a genetically obese mouse model of nonalcoholic steatohepatitis
An RNAi therapeutic targeting hepatic DGAT2 in a genetically obese mouse model of nonalcoholic steatohepatitis Open
View article: Structurally constrained phosphonate internucleotide linkage impacts oligonucleotide-enzyme interaction, and modulates siRNA activity and allele specificity
Structurally constrained phosphonate internucleotide linkage impacts oligonucleotide-enzyme interaction, and modulates siRNA activity and allele specificity Open
Oligonucleotides is an emerging class of chemically-distinct therapeutic modalities, where extensive chemical modifications are fundamental for their clinical applications. Inter-nucleotide backbones are critical to the behaviour of therap…
View article: Development of Novel Class of Therapeutic Oligonucleotides Based on Small Molecule Screening
Development of Novel Class of Therapeutic Oligonucleotides Based on Small Molecule Screening Open
Highly inefficient transit of oligonucleotides from outside cells to the intracellular compartments where functional activity of oligonucleotides takes place is the most serious limitation to the practical realization of a full potential o…
View article: 2′-O-Methyl at 20-mer Guide Strand 3′ Termini May Negatively Affect Target Silencing Activity of Fully Chemically Modified siRNA
2′-O-Methyl at 20-mer Guide Strand 3′ Termini May Negatively Affect Target Silencing Activity of Fully Chemically Modified siRNA Open
View article: Loss of huntingtin function slows synaptic vesicle endocytosis in striatal neurons from the httQ140/Q140 mouse model of Huntington's disease
Loss of huntingtin function slows synaptic vesicle endocytosis in striatal neurons from the httQ140/Q140 mouse model of Huntington's disease Open
View article: A CNS-Active siRNA Chemical Scaffold for the Treatment of Neurodegenerative Diseases
A CNS-Active siRNA Chemical Scaffold for the Treatment of Neurodegenerative Diseases Open
Small interfering RNAs (siRNAs) are a promising class of drugs for treating genetically-defined diseases. Therapeutic siRNAs enable specific modulation of gene expression, but require chemical architecture that facilitates efficient in viv…
View article: Serum Deprivation of Mesenchymal Stem Cells Improves Exosome Activity and Alters Lipid and Protein Composition
Serum Deprivation of Mesenchymal Stem Cells Improves Exosome Activity and Alters Lipid and Protein Composition Open
Exosomes can serve as delivery vehicles for advanced therapeutics. The components necessary and sufficient to support exosomal delivery have not been established. Here we connect biochemical composition and activity of exosomes to optimize…