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Publications

Peptide-conjugated antimiRs improve myotonic dystrophy type 1 phenotypes by promoting endogenous MBNL1 expression.

González-Martínez I. et al, (2023), Mol Ther Nucleic Acids, 34

Application of Antisense Conjugates for the Treatment of Myotonic Dystrophy Type 1.

Stoodley J. et al, (2023), Int J Mol Sci, 24

A modular RNA delivery system comprising spherical nucleic acids built on endosome-escaping polymeric nanoparticles

Garcia-Guerra A. et al, (2023), Nanoscale Advances

Peptide-Conjugated PMOs for the Treatment of Myotonic Dystrophy.

Stoodley J. et al, (2023), Methods Mol Biol, 2587, 209 - 237

CELL-PENETRATING PEPTIDE CONJUGATES AND METHODS OF THEIR USE

VARELA MA., (2022)

CELL-PENETRATING PEPTIDE CONJUGATES AND METHODS OF THEIR USE

VARELA MA., (2022)

Proof of concept of peptide-linked blockmiR-induced MBNL functional rescue in myotonic dystrophy type 1 mouse model.

Overby SJ. et al, (2022), Mol Ther Nucleic Acids, 27, 1146 - 1155

Preclinical characterization of antagomiR-218 as a potential treatment for myotonic dystrophy.

Cerro-Herreros E. et al, (2021), Mol Ther Nucleic Acids, 26, 174 - 191

Comparable Outcomes of Matched Sibling Donor and Matched Unrelated Donor Stem Cell Transplantation in Children With Acute Leukemia in Argentina.

Gómez SM. et al, (2021), J Pediatr Hematol Oncol

Application of CRISPR-Cas9-Mediated Genome Editing for the Treatment of Myotonic Dystrophy Type 1.

Marsh S. et al, (2020), Mol Ther, 28, 2527 - 2539

A Single Amino Acid Residue Regulates PTEN-Binding and Stability of the Spinal Muscular Atrophy Protein SMN.

Rademacher S. et al, (2020), Cells, 9

Linkers

VARELA MA., (2020)

Linkers

VARELA MA., (2020)

Cell-Penetrating peptides

RAZ R. et al, (2020)

Development of LNA Gapmer Oligonucleotide-Based Therapy for ALS/FTD Caused by the C9orf72 Repeat Expansion.

Sathyaprakash C. et al, (2020), Methods Mol Biol, 2176, 185 - 208

Peptide-conjugated oligonucleotides evoke long-lasting myotonic dystrophy correction in patient-derived cells and mice.

Klein AF. et al, (2019), J Clin Invest

Proteomic evaluation of Pip6a-PMO treatment for myotonic dystrophy type 1

Holland A. et al, (2018), NEUROMUSCULAR DISORDERS, 28, S8 - S9

Knockdown and replacement therapy mediated by artificial mirtrons in spinocerebellar ataxia 7.

Curtis HJ. et al, (2017), Nucleic Acids Res, 45, 7870 - 7885

Functional Delivery of Lipid-Conjugated siRNA by Extracellular Vesicles.

O'Loughlin AJ. et al, (2017), Mol Ther, 25, 1580 - 1587

C9orf72 and RAB7L1 regulate vesicle trafficking in amyotrophic lateral sclerosis and frontotemporal dementia.

Aoki Y. et al, (2017), Brain, 140, 887 - 897

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