Partial correction of the dwarf phenotype by non-viral transfer of the growth hormone gene in mice: Treatment age is critical

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Partial correction of the dwarf phenotype by non-viral transfer of the growth hormone gene in mice : Treatment age is critical. / Higuti, Eliza; Cecchi, Cláudia R; Oliveira, Nélio A J; Lima, Eliana R; Vieira, Daniel P; Aagaard, Lars; Jensen, Thomas G; Jorge, Alexander A L; Bartolini, Paolo; Peroni, Cibele N.

I: Growth Hormone & I G F Research, Bind 26, 02.2016, s. 1-7.

Publikation: Bidrag til tidsskrift/Konferencebidrag i tidsskrift /Bidrag til avisTidsskriftartikelForskningpeer review

Harvard

Higuti, E, Cecchi, CR, Oliveira, NAJ, Lima, ER, Vieira, DP, Aagaard, L, Jensen, TG, Jorge, AAL, Bartolini, P & Peroni, CN 2016, 'Partial correction of the dwarf phenotype by non-viral transfer of the growth hormone gene in mice: Treatment age is critical', Growth Hormone & I G F Research, bind 26, s. 1-7. https://doi.org/10.1016/j.ghir.2015.12.001

APA

Higuti, E., Cecchi, C. R., Oliveira, N. A. J., Lima, E. R., Vieira, D. P., Aagaard, L., ... Peroni, C. N. (2016). Partial correction of the dwarf phenotype by non-viral transfer of the growth hormone gene in mice: Treatment age is critical. Growth Hormone & I G F Research, 26, 1-7. https://doi.org/10.1016/j.ghir.2015.12.001

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Vancouver

Author

Higuti, Eliza ; Cecchi, Cláudia R ; Oliveira, Nélio A J ; Lima, Eliana R ; Vieira, Daniel P ; Aagaard, Lars ; Jensen, Thomas G ; Jorge, Alexander A L ; Bartolini, Paolo ; Peroni, Cibele N. / Partial correction of the dwarf phenotype by non-viral transfer of the growth hormone gene in mice : Treatment age is critical. I: Growth Hormone & I G F Research. 2016 ; Bind 26. s. 1-7.

Bibtex

@article{28704fc55ef041379b13fcb96a8f141b,
title = "Partial correction of the dwarf phenotype by non-viral transfer of the growth hormone gene in mice: Treatment age is critical",
abstract = "Non-viral transfer of the growth hormone gene to different muscles of immunodeficient dwarf (lit/scid) mice is under study with the objective of improving phenotypic correction via this particular gene therapy approach. Plasmid DNA was administered into the exposed quadriceps or non-exposed tibialis cranialis muscle of lit/scid mice followed by electroporation, monitoring several growth parameters. In a 6-month bioassay, 50μg DNA were injected three times into the quadriceps muscle of 80-day old mice. A 50{\%} weight increase, with a catch-up growth of 21{\%}, together with a 16{\%} increase for nose-to-tail and tail lengths (catch-up=19-21{\%}) and a 24-28{\%} increase for femur length (catch-up=53-60{\%}), were obtained. mIGF1 serum levels were ~7-fold higher than the basal levels for untreated mice, but still ~2-fold lower than in non-dwarf scid mice. Since treatment age was found to be particularly important in a second bioassay utilizing 40-day old mice, these pubertal mice were compared in a third bioassay with adult (80-day old) mice, all treated twice with 50μg DNA injected into each tibialis cranialis muscle, via a less invasive approach. mIGF1 concentrations at the same level as co-aged scid mice were obtained 15days after administration in pubertal mice. Catch-up growth, based on femur length (77{\%}), nose-to-tail (36{\%}) and tail length (39{\%}) increases was 40 to 95{\%} higher than those obtained upon treating adult mice. These data pave the way for the development of more effective pre-clinical assays in pubertal dwarf mice for the treatment of GH deficiency via plasmid-DNA muscular administration.",
author = "Eliza Higuti and Cecchi, {Cl{\'a}udia R} and Oliveira, {N{\'e}lio A J} and Lima, {Eliana R} and Vieira, {Daniel P} and Lars Aagaard and Jensen, {Thomas G} and Jorge, {Alexander A L} and Paolo Bartolini and Peroni, {Cibele N}",
note = "Copyright {\circledC} 2015 Elsevier Ltd. All rights reserved.",
year = "2016",
month = "2",
doi = "10.1016/j.ghir.2015.12.001",
language = "English",
volume = "26",
pages = "1--7",
journal = "Growth Hormone & I G F Research",
issn = "1096-6374",
publisher = "Churchill Livingstone",

}

RIS

TY - JOUR

T1 - Partial correction of the dwarf phenotype by non-viral transfer of the growth hormone gene in mice

T2 - Treatment age is critical

AU - Higuti, Eliza

AU - Cecchi, Cláudia R

AU - Oliveira, Nélio A J

AU - Lima, Eliana R

AU - Vieira, Daniel P

AU - Aagaard, Lars

AU - Jensen, Thomas G

AU - Jorge, Alexander A L

AU - Bartolini, Paolo

AU - Peroni, Cibele N

N1 - Copyright © 2015 Elsevier Ltd. All rights reserved.

PY - 2016/2

Y1 - 2016/2

N2 - Non-viral transfer of the growth hormone gene to different muscles of immunodeficient dwarf (lit/scid) mice is under study with the objective of improving phenotypic correction via this particular gene therapy approach. Plasmid DNA was administered into the exposed quadriceps or non-exposed tibialis cranialis muscle of lit/scid mice followed by electroporation, monitoring several growth parameters. In a 6-month bioassay, 50μg DNA were injected three times into the quadriceps muscle of 80-day old mice. A 50% weight increase, with a catch-up growth of 21%, together with a 16% increase for nose-to-tail and tail lengths (catch-up=19-21%) and a 24-28% increase for femur length (catch-up=53-60%), were obtained. mIGF1 serum levels were ~7-fold higher than the basal levels for untreated mice, but still ~2-fold lower than in non-dwarf scid mice. Since treatment age was found to be particularly important in a second bioassay utilizing 40-day old mice, these pubertal mice were compared in a third bioassay with adult (80-day old) mice, all treated twice with 50μg DNA injected into each tibialis cranialis muscle, via a less invasive approach. mIGF1 concentrations at the same level as co-aged scid mice were obtained 15days after administration in pubertal mice. Catch-up growth, based on femur length (77%), nose-to-tail (36%) and tail length (39%) increases was 40 to 95% higher than those obtained upon treating adult mice. These data pave the way for the development of more effective pre-clinical assays in pubertal dwarf mice for the treatment of GH deficiency via plasmid-DNA muscular administration.

AB - Non-viral transfer of the growth hormone gene to different muscles of immunodeficient dwarf (lit/scid) mice is under study with the objective of improving phenotypic correction via this particular gene therapy approach. Plasmid DNA was administered into the exposed quadriceps or non-exposed tibialis cranialis muscle of lit/scid mice followed by electroporation, monitoring several growth parameters. In a 6-month bioassay, 50μg DNA were injected three times into the quadriceps muscle of 80-day old mice. A 50% weight increase, with a catch-up growth of 21%, together with a 16% increase for nose-to-tail and tail lengths (catch-up=19-21%) and a 24-28% increase for femur length (catch-up=53-60%), were obtained. mIGF1 serum levels were ~7-fold higher than the basal levels for untreated mice, but still ~2-fold lower than in non-dwarf scid mice. Since treatment age was found to be particularly important in a second bioassay utilizing 40-day old mice, these pubertal mice were compared in a third bioassay with adult (80-day old) mice, all treated twice with 50μg DNA injected into each tibialis cranialis muscle, via a less invasive approach. mIGF1 concentrations at the same level as co-aged scid mice were obtained 15days after administration in pubertal mice. Catch-up growth, based on femur length (77%), nose-to-tail (36%) and tail length (39%) increases was 40 to 95% higher than those obtained upon treating adult mice. These data pave the way for the development of more effective pre-clinical assays in pubertal dwarf mice for the treatment of GH deficiency via plasmid-DNA muscular administration.

U2 - 10.1016/j.ghir.2015.12.001

DO - 10.1016/j.ghir.2015.12.001

M3 - Journal article

VL - 26

SP - 1

EP - 7

JO - Growth Hormone & I G F Research

JF - Growth Hormone & I G F Research

SN - 1096-6374

ER -