Recent Papers from the Gait group

2017

Solid phase synthesis of long and difficult PNAs through selective Hmb incorporation: Application to the total synthesis of peptide-PNAs.
J. Tailhades, H. Takizawa, M. J. Gait, J. D. Wade, A. Yoshitsugu and F. Shabanpoor.
Amino Acids (2017).
https://doi.org/10.3389/fchem.2017.00081

Identification of a peptide for systemic brain delivery of a morpholino oligonucleotide in mouse models of spinal muscular atrophy.
F. Shabanpoor, S. M. Hammond, F. Abendroth, G. Hazell, M.J.A. Wood and M.J. Gait.
Nucleic Acids Therapeutics (2017) 27, 130-143.

ClickIn: a flexible protocol for quantifying mitochondrial uptake of nucleobase derivatives.
K. Hoogewijs, A.M. James, R.A.J. Smith, F. Abendroth, M.J. Gait, M.P. Murphy and R.N. Lightowlers.
Interface Focus (2017) 7, 20160117.
https://dx.doi.org/10.1098/rsfs.2016.0117

2016

Systemic peptide-mediated, oligonucleotide therapy improves long-term survival in spinal muscular atrophy.
S. M. Hammond, G. Hazell, F. Shabanpoor, A. F. Saleh, M. Bowerman, J. N. Sleigh, K. M. Meijboom, H. Zhou, F. Muntoni, K. Talbot, M. J. Gait and M. J. A. Wood.
Proc. Natl. Acad. Sci. USA (2016) 113, 10962-10967

273. Assessing the delivery of mitochondria-targeted molecules using Click chemistry.
K. Hoogewijs, A.M. James R.A.J. Smith, M.J. Gait, M.P. Murphy and R. Lightowlers.
ChemBioChem. (2016) 17, 1312-1316.

2015

Self-assembly and receptor mediated uptake of therapeutic antisense oligonucleotides.
K. Ezzat, Y. Aoki, T. Koo, A. Coenen-Stass, G. McClorey, L. Benner, L. O'Donovan, A.F. Saleh, M. Behlke, J. Morris, A. Goyenvalle, C. Leumann, S. El Andaloussi, S. Gordon, M.J. Gait and M.J.A. Wood.
Nano Letters (2015) 15, 4364-4373.

Multi-level omics analysis of gene expression in a murine model of dystrophin loss and therapeutic restoration.
T.C. Roberts, H.J. Johansson, G. McClorey, C. Godfrey, K.E.M. Blomberg, T. Coursindel, M.J. Gait, C.I.E. Smith, J. Lehto, S. EL Andaloussi and M.J.A. Wood.
Hum. Mol. Gen. (2015) 24, 6756-6768.

Identification of novel, therapy-responsive protein biomarkers for Duchenne muscular dystrophy by aptamer-based serum proteomics.
Coenen-Stass, A.M., McClorey, G., Manzano, R., Betts, C.A., Blain, A. Saleh, A.F., Gait, M.J., Lochmuller, H., Wood, M.J.A. and Roberts, T.C.
Scientific Reports (2015) 5: 17014.

Combination antisense treatment for Duchenne muscular dystrophy: open reading frame rescue of dystrophin in conjunction with destructive exon skipping of myostatin in neonatal mdx mice.
N.B. Lu-Nguyen, S.A. Jarmin, A.F. Saleh, L. Popplewell, M.J. Gait and G. Dickson.
Molecular Therapy (2015) 23, 1341-1348.

How much dystrophin is enough: defining dystrophin levels for clinical efficacy in DMD.
C. Godfrey, S. Muses, G. McClorey, K. Wells, T. Coursindel, R. Terry, C. Betts, S. Hammond, L. O'Donovan, J. Hildyard, S. EL Andaloussi, M.J. Gait, M.J.A. Wood and D.J. Wells.
Human Mol. Gen. (2015) 24, 4225-4237.

Prevention of exercised induced cardiomyopathy following Pip-PMO treatment in dystrophic mdx mice.
C.A. Betts, A.F. Saleh, C.A. Carr, S.M. Hammond, A.M.L. Coenen-Stass, C. Godfrey, G. McClorey, M.A. Varela, T.C. Roberts, K. Clarke, M.J. Gait and M.J.A. Wood.
Scientific Reports, (2015) 5:8986.

Peptide nanoparticle delivery of charge-neutral splice switching morpholino oligonucleotides.
P. Järver, E. M. Zaghloul, A.A. Arzumanov, A. Saleh, G. McClorey, S.M. Hammond, M. Hällbrink, Ü. Langel, C. I. E. Smith, M. J. A. Wood, M.J. Gait and S. El Andaloussi.
Nucleic Acids Therapeutics (2015), 25, 65-77.

Delivery of therapeutic oligonucleotides with cell-penetrating peptides.
P. Boisguerin, S. Deshayes, M.J. Gait. L. O’Donovan, C. Godfrey, C.A. Betts, M.J.A. Wood and B. Lebleu.
Adv. Drug Delivery Rev. (2015) 87, 52-67.

Bi-specific splice-switching PMO oligonucleotides conjugated via a single peptide active in a mouse model of Duchenne muscular dystrophy.
F. Shabanpoor, G. McClorey, P. Järver, A. Saleh, M.J.A. Wood and M.J. Gait,
Nucleic Acids Res. (2015) 43, 29-39.

2014

Splice-correcting oligonucleotides restore BTK function in X-linked agammaglobulinemia model.
B. Bestas, P.M.D. Moreno, K.E.M. Blomberg, D.K. Mohammad, A.F. Saleh, T. Sutlu, P. Guterstam, M.O. Gustafsson, B.Piątosa, T.C. Roberts, M.A. Behlke, M.J.A. Wood, M.J. Gait, K.E. Lundin, S. EL-Andaloussi1, R. Månsson, A. Berglöf , J. Wengel, C.I.E. Smith,
J. Clin. Investigation (2014) 124, 4067-4081.

Parallel synthesis of cell-penetrating peptide conjugates of PMO towards exon skipping enhancement in Duchenne muscular dystrophy.
L. O’Donovan, I. Okamoto, A. A. Arzumanov. D.L. Williams. P. Deuss and M.J. Gait
Nucleic Acids Therapeutics (2014) 25, 1-10.

Cellular trafficking determines the exon skipping activity of Pip6a-PMOin mdx skeletal and cardiac muscle cells.
T. Lehto, A. Castillo Alvaraz, M.J. Gait, T. Coursindel, M.J.A. Wood, B. Lebleu and P. Boisguerin
Nucleic Acids Res. (2014) 42, 3207-3217.

A chemical view of oligonucleotides for exon skipping and related drug applications.
P. Järver, L. O’Donovan and M.J. Gait
Nucleic Acids Therapeutics (2014) 24, 37-47.

Synthetic microRNA blocking agents.
P. Järver, A.G. Torres and M.J. Gait
Applied RNAi. From Fundamental Research to Therapeutic Applications (2014)) (P. Arbuthnot and M. S. Weinberg eds.), Caister Academic Press, Norfolk, UK, pp 105-126.

2013

Parallel synthesis and splicing redirection activity of cell-penetrating peptide conjugate libraries of a PNA cargo.
P.J. Deuss, A.A Arzumanov, D.L. Williams and M.J. Gait,
Org. Biomol. Chem. (2013) 11, 7621-7630.

Development of a general methodology for labelling peptide conjugates of morpholino (PMO) oligonucleotides using alkyne-azide click chemistry.
F. Shabanpoor and M.J. Gait,
Chem. Commun. (2013) 49, 10260-10262.

Enhanced splice correction by 3′, 5′-serinol and 2′-(ω-O-methylserinol) guarded OMe-RNA/DNA mixmers in cells.
V. Kotikam, A. Arzumanov, M. J. Gait and V. Kumar,
Artificial DNA, PNA and XNA (2013) 4:3, 1-7.

2012

Synthesis and biological activity of phosphonoacetate and thiophosphonoacetate modified 2’-O-methyl oligoribonucleotides.
R. N. Threlfall, A. G. Torres, A Krivenko, M. J. Gait and M. H. Caruthers,
Org. Biomol. Chem., (2012) 10, 746-754.

245. Chemical structure requirements and cellular targeting of microRNA-122 by peptide nucleic acids anti-miRs.
A.G. Torres, M.M. Fabani, E. Vigorito, D. Williams, N. Al-Obaidi, F. Wojciechowski, R.H.E. Hudson, O. Seitz and M.J. Gait,
Nucleic Acids Res. (2012) 40, 2152-2167.

Exploiting cell surface thiols to enhance cellular uptake
A.G. Torres and M.J. Gait,
Trends Biotech. (2012) 30, 185-190.

Peptide-based in vivo delivery agents for oligonucleotides and siRNA,
T. Coursindel, P. Järver and M.J. Gait,
Nucleic Acids Ther. (2012) 22, 71-76.

Pip6-PMO, a new generation of peptide-oligonucleotide conjugates with improved cardiac exon skipping activity for Duchenne muscular dystrophy treatment
C. Betts, A.F. Saleh, A.A. Arzumanov, S.M. Hammond, C. Godfrey, T. Coursindel, M.J. Gait and M.J.A.Wood,
Molecular Therapy Nucleic Acids (2012) 1, e38.

Peptide-mediated cell and in vivo delivery of antisense oligonucleotides and siRNA.
P. Järver, T. Coursindel, S. EL Andaloussi, C. Godfrey, M.J.A. Wood, and M. J. Gait,
Molecular Therapy Nucleic Acids (2012) 1, e27

Expression analysis in multiple muscle groups and serum reveals complexity in the microRNA transcriptome of the mdx mouse and implications for therapy.
T.C. Roberts, K.E.M. Blomberg, G. McClorey, S.EL Andaloussi, C. Godfrey, C. Betts, T. Coursindel, M. J. Gait, C.I.E. Smith and M.J.A. Wood,
Molecular Therapy Nucleic Acids (2012) 1, e39.

Dual myostatin and dystrophin exon skipping by morpholino nucleic acid oligomers conjugated to a cell penetrating peptide is a promising therapeutic strategy for the treatment of Duchenne muscular dystrophy.
A Malerba, J.K Kang, G. McClorey, G., A.F. Saleh, L. Popplewell, M.J. Gait, M.J.A. Wood and G. Dickson,
Molecular Therapy Nucleic Acids (2012) 1, e62.

Overview of alternative oligonucleotide chemistries for exon skipping.
A.F. Saleh, A.A. Arzumanov and M.J. Gait,
Methods in Molecular Biology: Exon Skipping, (2012) 867, 365-378.