Alexander McCampbell
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Explore the profile of Alexander McCampbell including associated specialties, affiliations and a list of published articles.
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25
Citations
1536
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Recent Articles
1.
Chen Y, Kankel M, Hana S, Lau S, Zavodszky M, McKissick O, et al.
Gene Ther
. 2022 Nov;
30(5):443-454.
PMID: 36450833
CRISPR-based gene editing technology represents a promising approach to deliver therapies for inherited disorders, including amyotrophic lateral sclerosis (ALS). Toxic gain-of-function superoxide dismutase 1 (SOD1) mutations are responsible for ~20%...
2.
Paris A, Bora P, Parolo S, Monine M, Tong X, Eraly S, et al.
CPT Pharmacometrics Syst Pharmacol
. 2022 Feb;
11(4):447-457.
PMID: 35146969
Neurofilaments (Nfs) are the major structural component of neurons. Their role as a potential biomarker of several neurodegenerative diseases has been investigated in past years with promising results. However, even...
3.
Fumagalli L, Young F, Boeynaems S, De Decker M, Mehta A, Swijsen A, et al.
Sci Adv
. 2021 Apr;
7(15).
PMID: 33837088
A hexanucleotide repeat expansion in the gene is the most common genetic cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). How this mutation leads to these neurodegenerative diseases...
4.
Torregrosa T, Lehman S, Hana S, Marsh G, Xu S, Koszka K, et al.
Gene Ther
. 2021 Feb;
28(7-8):456-468.
PMID: 33612827
Adeno-associated virus (AAV) transduction efficiency and tropism are conventionally determined by high expression of a fluorescent reporter gene. Emerging data has suggested that such conventional methods may underestimate AAV transduction...
5.
Cook C, Wu Y, Odeh H, Gendron T, Jansen-West K, Del Rosso G, et al.
Sci Transl Med
. 2020 Sep;
12(559).
PMID: 32878979
TAR DNA-binding protein 43 (TDP-43) inclusions are a pathological hallmark of frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS), including cases caused by GC repeat expansions in the gene (c9FTD/ALS)....
6.
Miller T, Cudkowicz M, Shaw P, Andersen P, Atassi N, Bucelli R, et al.
N Engl J Med
. 2020 Jul;
383(2):109-119.
PMID: 32640130
Background: Tofersen is an antisense oligonucleotide that mediates the degradation of superoxide dismutase 1 (SOD1) messenger RNA to reduce SOD1 protein synthesis. Intrathecal administration of tofersen is being studied for...
7.
Kankel M, Sen A, Lu L, Theodorou M, Dimlich D, McCampbell A, et al.
Genetics
. 2020 Apr;
215(3):747-766.
PMID: 32345615
Amyotrophic lateral sclerosis (ALS), commonly known as Lou Gehrig's disease, is a devastating neurodegenerative disorder lacking effective treatments. ALS pathology is linked to mutations in >20 different genes indicating a...
8.
Cammack A, Atassi N, Hyman T, van den Berg L, Harms M, Baloh R, et al.
Neurology
. 2019 Oct;
93(17):e1605-e1617.
PMID: 31578300
Objective: To define the natural history of the amyotrophic lateral sclerosis (C9ALS) patient population, develop disease biomarkers, and characterize patient pathologies. Methods: We prospectively collected clinical and demographic data from...
9.
Darras B, Crawford T, Finkel R, Mercuri E, De Vivo D, Oskoui M, et al.
Ann Clin Transl Neurol
. 2019 May;
6(5):932-944.
PMID: 31139691
Objective: To evaluate plasma phosphorylated neurofilament heavy chain (pNF-H) as a biomarker in spinal muscular atrophy (SMA). Methods: Levels of pNF-H were measured using the ProteinSimple platform in plasma samples...
10.
Zhang Y, Guo L, Gonzales P, Gendron T, Wu Y, Jansen-West K, et al.
Science
. 2019 Feb;
363(6428).
PMID: 30765536
How hexanucleotide GGGGCC (GC) repeat expansions in cause frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS) is not understood. We developed a mouse model engineered to express poly(PR), a proline-arginine...