Christian Hinderer
Overview
Explore the profile of Christian Hinderer including associated specialties, affiliations and a list of published articles.
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26
Citations
1333
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Recent Articles
1.
Rosenberg J, Fung E, Dyke J, De B, Lou H, Kelly J, et al.
Hum Gene Ther
. 2023 Aug;
34(21-22):1095-1106.
PMID: 37624734
Based on studies in experimental animals demonstrating that administration of adeno-associated virus (AAV) vectors to the cerebrospinal fluid (CSF) is an effective route to transfer genes to the nervous system,...
2.
Blair I, Rojsajjakul T, Hordeaux J, Chaudhary G, Hinderer C, Mesaros C, et al.
Res Sq
. 2023 Jul;
PMID: 37461697
Deficiency in human mature frataxin (hFXN-M) protein is responsible for the devastating neurodegenerative and cardiodegenerative disease of Friedreich's ataxia (FRDA). It results primarily by epigenetic silencing the FXN gene due...
3.
Sims J, Greig J, Michalson K, Lian S, Martino R, Meggersee R, et al.
PLoS Pathog
. 2021 Jul;
17(7):e1009544.
PMID: 34265018
SARS-CoV-2 variants have emerged with enhanced pathogenicity and transmissibility, and escape from pre-existing immunity, suggesting first-generation vaccines and monoclonal antibodies may now be less effective. Here we present an approach...
4.
Hinderer C, Nosratbakhsh B, Katz N, Wilson J
Hum Gene Ther
. 2020 Oct;
31(21-22):1169-1177.
PMID: 33045869
GM1 gangliosidosis is a rare neurodegenerative lysosomal storage disease caused by loss-of-function mutations in the gene encoding beta-galactosidase (β-gal). There are no approved treatments for GM1 gangliosidosis. Previous studies in...
5.
Hinderer C, Miller R, Dyer C, Johansson J, Bell P, Buza E, et al.
Ann Clin Transl Neurol
. 2020 Sep;
7(10):1843-1853.
PMID: 32937039
Objective: Dominant loss-of-function mutations in the gene encoding the lysosomal protein, progranulin, cause 5-10% of frontotemporal dementia cases. As progranulin undergoes secretion and endocytosis, a small number of progranulin-expressing cells...
6.
Hordeaux J, Buza E, Dyer C, Goode T, Mitchell T, Richman L, et al.
Hum Gene Ther
. 2020 Aug;
31(15-16):808-818.
PMID: 32845779
The administration of adeno-associated virus (AAV) vectors to nonhuman primates (NHP) via the blood or cerebrospinal fluid (CSF) can lead to dorsal root ganglion (DRG) pathology. The pathology is minimal...
7.
Hinderer C, Katz N, Dyer C, Goode T, Johansson J, Bell P, et al.
Mol Ther Methods Clin Dev
. 2020 May;
17:969-974.
PMID: 32420410
Preclinical studies have demonstrated that a single injection of an adeno-associated virus (AAV) vector into the cerebrospinal fluid (CSF) can achieve widespread gene transfer throughout the central nervous system. Successfully...
8.
Hordeaux J, Hinderer C, Buza E, Louboutin J, Jahan T, Bell P, et al.
Hum Gene Ther
. 2019 Apr;
30(8):957-966.
PMID: 31017018
Many neuropathic diseases cause early, irreversible neurologic deterioration, which warrants therapeutic intervention during the first months of life. In the case of mucopolysaccharidosis type I, a recessive lysosomal storage disorder...
9.
Hordeaux J, Hinderer C, Goode T, Katz N, Buza E, Bell P, et al.
Mol Ther Methods Clin Dev
. 2018 Aug;
10:79-88.
PMID: 30073179
Mucopolysaccharidosis type I is a recessive genetic disease caused by deficiency of the lysosomal enzyme α-L-iduronidase, which leads to a neurodegenerative and systemic disease called Hurler syndrome in its most...
10.
Hordeaux J, Hinderer C, Goode T, Buza E, Bell P, Calcedo R, et al.
Mol Ther Methods Clin Dev
. 2018 Aug;
10:68-78.
PMID: 30073178
Hunter syndrome is an X-linked recessive disease caused by deficiency of the lysosomal enzyme iduronate-2-sulfatase. The severe form of this progressive, systemic, and neurodegenerative disease results in loss of cognitive...