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ey0020.2-10 | Long-Acting Growth Hormone (LAGH) | ESPEYB20

2.10. Long-acting PEGylated growth hormone in children with idiopathic short stature

X Luo , S Zhao , Y Yang , G Dong , L Chen , P Li , F Luo , C Gong , Z Xu , X Xu , H Gong , H Du , L Hou , Y Zhong , Q Shi , X Chen , X Chen , L Xu , R Cheng , C Su , Y Ma , L Xu , L Zhang , H Lu

Brief summary: This randomized, multicenter, controlled, phase II study compared the effects of high-dose (HD) once-weekly PEGylated-recombinant human growth hormone (PEG-rhGH) to low-dose (LD) and to an untreated control group of children with idiopathic short stature (ISS) over a period of 52 weeks. PEG-rhGH was effective in increasing height gain in a dose dependent manner with both doses being well tolerated during the observation period.PEG-rhGH is ...

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ey0015.14-10 | CRISPR-Cas9 gene therapy | ESPEYB15

14.10 Correction of a pathogenic gene mutation in human embryos

H Ma , N Marti-Gutierrez , SW Park , J Wu , Y Lee , K Suzuki , A Koski , D Ji , T Hayama , R Ahmed , H Darby , C Van Dyken , Y Li , E Kang , AR Park , D Kim , ST Kim , J Gong , Y Gu , X Xu , D Battaglia , SA Krieg , DM Lee , DH Wu , DP Wolf , SB Heitner , JCI Belmonte , P Amato , JS Kim , S Kaul , S Mitalipov

To read the full abstract: Nature 2017;548:413-419Over recent years, the Yearbook has followed the rapid advances in CRISPR-Cas9 gene editing technology, initially as a widely adopted research tool, but also as an emerging form of gene therapy. Here, Ma et al. report the first use of CRISPR–Cas9 to efficiently and safely correct a pathogenic heterozygous mutation in human embryos. The...

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ESPE Yearbook of Paediatric Endocrinology

2.10. Long-acting PEGylated growth hormone in children with idiopathic short stature

14.10 Correction of a pathogenic gene mutation in human embryos

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