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Early-childhood linear growth faltering in low- and middle-income countries
A pooled analysis of longitudinal studies in low- and middle-income countries identifies the typical age of onset of linear growth faltering and investigates recurrent faltering in early life.
- Jade Benjamin-Chung
- , Andrew Mertens
- & Pablo Penataro Yori
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Article
| Open Access
A framework for individualized splice-switching oligonucleotide therapy
Whole-genome sequencing analyses in a cohort of individuals with ataxia-telangiectasia are used to identify genetic variants that might be amenable to treatment with splice-switching antisense oligonucleotides (ASOs), and develop ASOs with therapeutic potential.
- Jinkuk Kim
- , Sijae Woo
- & Timothy W. Yu
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Article
| Open Access
Genomic investigations of unexplained acute hepatitis in children
An investigation using various methods reports an association between adeno-associated virus 2 and paediatric hepatitis of unknown aetiology.
- Sofia Morfopoulou
- , Sarah Buddle
- & Judith Breuer
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Article
| Open Access
Diminishing benefits of urban living for children and adolescents’ growth and development
The advantage of living in cities compared with rural areas with respect to height and BMI in children and adolescents has generally become smaller globally from 1990 to 2020, except in sub-Saharan Africa.
- Anu Mishra
- , Bin Zhou
- & Majid Ezzati
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Article
| Open Access
GD2-CAR T cell therapy for H3K27M-mutated diffuse midline gliomas
A phase I dose-escalation trial of GD2-CAR T cells in children and young adults with diffuse midline gliomas to assess the feasibility of manufacturing, safety and tolerability, and to preliminarily assess efficacy.
- Robbie G. Majzner
- , Sneha Ramakrishna
- & Michelle Monje
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Letter
| Open Access
The human gut microbiome in early-onset type 1 diabetes from the TEDDY study
An analysis of more than 10,000 metagenomes from the TEDDY study provides a detailed functional profile of the gut microbiome in relation to islet autoimmunity, and supports the protective effects of short-chain fatty acids in early-onset type 1 diabetes.
- Tommi Vatanen
- , Eric A. Franzosa
- & Ramnik J. Xavier
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Letter |
Neonatal selection by Toll-like receptor 5 influences long-term gut microbiota composition
Age-dependent epithelial expression of the innate immune receptor TLR5 in the gut of newborn mice selects against the presence of flagellated bacteria and influences microbiota composition throughout life.
- Marcus Fulde
- , Felix Sommer
- & Mathias W. Hornef
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Brief Communications Arising |
Lens regeneration in children
- Demetrios G. Vavvas
- , Thaddeus P. Dryja
- & Scott R. Lambert
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Brief Communications Arising |
Improving outcomes in congenital cataract
- Ameenat Lola Solebo
- , Christopher J. Hammond
- & Jugnoo S. Rahi
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Article |
Statin treatment rescues FGFR3 skeletal dysplasia phenotypes
This study reprograms fibroblasts from thanatophoric dysplasia type I (TD1) and achondroplasia (ACH) patients into induced pluripotent stem cells (iPSCs), finding that chondrogenic differentiation results in the formation of degraded cartilage; statin treatment led to significant recovery of bone growth in a mouse model of ACH.
- Akihiro Yamashita
- , Miho Morioka
- & Noriyuki Tsumaki
Child wasting and concurrent stunting in low- and middle-income countries