Fazel Shabanpoor

Endosomal escape cell-penetrating peptides significantly enhance pharmacological effectiveness and CNS activity of systemically administered antisense oligonucleotides

Mohadeseh Dastpeyman, Ramin Sharifi, Azin Amin, John A Karas, Brittany Cuic, Yijun Pan, Joseph A Nicolazzo, Bradley J Turner, Fazel Shabanpoor

Journal article  |  2021  |  INTERNATIONAL JOURNAL OF PHARMACEUTICS

Antisense oligonucleotides (ASOs) are an emerging class of gene-specific therapeutics for diseases associated with the central ner..

Modular Synthesis of Trifunctional Peptide-oligonucleotide Conjugates via Native Chemical Ligation

Mohadeseh Dastpeyman, John A Karas, Azin Amin, Bradley J Turner, Fazel Shabanpoor

Journal article  |  2021  |  FRONTIERS IN CHEMISTRY

Cell penetrating peptides (CPPs) are being increasingly used as efficient vectors for intracellular delivery of biologically activ..

Amyotrophic Lateral Sclerosis and Autophagy: Dysfunction and Therapeutic Targeting

Azin Amin, Nirma D Perera, Philip M Beart, Bradley J Turner, Fazel Shabanpoor

Journal article  |  2020  |  CELLS

Over the past 20 years, there has been a drastically increased understanding of the genetic basis of Amyotrophic Lateral Sclerosis..

Application of Urine-Derived Stem Cells to Cellular Modeling in Neuromuscular and Neurodegenerative Diseases

Mitsuto Sato, Hotake Takizawa, Akinori Nakamura, Bradley J Turner, Fazel Shabanpoor, Yoshitsugu Aoki

Journal article  |  2019  |  FRONTIERS IN MOLECULAR NEUROSCIENCE

Neuromuscular and neurodegenerative diseases are mostly modeled using genetically modified animals such as mice. However, animal m..

Thiol-Cyanobenzothiazole Ligation for the Efficient Preparation of Peptide-PNA Conjugates

Nitin A Patil, John A Karas, Bradley J Turner, Fazel Shabanpoor

Journal article  |  2019  |  BIOCONJUGATE CHEMISTRY

Antisense oligonucleotide (ASO)-based drugs are emerging with great potential as therapeutic compounds for diseases with unmet med..

Scavenger Receptor Class A1 Mediates Uptake of Morpholino Antisense Oligonucleotide into Dystrophic Skeletal Muscle

Shouta Miyatake, Yoshitaka Mizobe, Maria K Tsoumpra, Kenji Rowel Q Lim, Yuko Hara, Fazel Shabanpoor, Toshifumi Yokota, Shin'ichi Takeda, Yoshitsugu Aoki

Journal article  |  2019  |  MOLECULAR THERAPY-NUCLEIC ACIDS

Exon skipping using phosphorodiamidate morpholino oligomers (PMOs) is a promising treatment strategy for Duchenne muscular dystrop..

The Assembly of Fluorescently Labeled Peptide-Oligonucleotide Conjugates via Orthogonal Ligation Strategies

John Karas, Bradley J Turner, Fazel Shabanpoor

Book Chapter  |  2018  |  Methods in Molecular Biology

Efficient intracellular delivery is critical to the successful application of synthetic antisense oligonucleotides (ASOs) to modul..

Amphiphilic lipopeptide significantly enhances uptake of charge-neutral splice switching morpholino oligonucleotide in spinal muscular atrophy patient-derived fibroblasts

Behnoosh Tajik-Ahmadabad, Anastasios Polyzos, Frances Separovic, Fazel Shabanpoor

Journal article  |  2017  |  INTERNATIONAL JOURNAL OF PHARMACEUTICS

Splice-switching antisense oligonucleotides (SSOs) are emerging therapeutics with two SSOs recently approved by the FDA for Duchen..

Solid-Phase Synthesis of Difficult Purine-Rich PNAs through Selective Hmb Incorporation: Application to the Total Synthesis of Cell Penetrating Peptide-PNAs

Julien Tailhades, Hotake Takizawa, Michael J Gait, Don A Wellings, John D Wade, Yoshitsugu Aoki, Fazel Shabanpoor

Journal article  |  2017  |  FRONTIERS IN CHEMISTRY

Antisense oligonucleotide (ASO)-based drug development is gaining significant momentum following the recent FDA approval of Etepli..

Combination of valproic acid and morpholino splice-switching oligonucleotide produces improved outcomes in spinal muscular atrophy patient-derived fibroblasts

Anna Farrelly-Rosch, Chew Ling Lau, Nitin Patil, Bradley J Turner, Fazel Shabanpoor

Journal article  |  2017  |  NEUROCHEMISTRY INTERNATIONAL

Spinal muscular atrophy (SMA), the leading genetic cause of infant mortality worldwide, is characterised by the homozygous loss of..