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Year Number of Results
1970 1
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1989 1
1991 2
1993 3
1994 3
1995 2
1997 2
1998 3
1999 7
2000 6
2001 2
2002 4
2003 8
2004 2
2005 8
2006 7
2007 4
2008 1
2009 1
2010 2
2011 2
2012 6
2013 5
2014 4
2015 2
2016 4
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2025 0

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104 results

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Page 1
Current Clinical Applications of In Vivo Gene Therapy with AAVs.
Mendell JR, Al-Zaidy SA, Rodino-Klapac LR, Goodspeed K, Gray SJ, Kay CN, Boye SL, Boye SE, George LA, Salabarria S, Corti M, Byrne BJ, Tremblay JP. Mendell JR, et al. Mol Ther. 2021 Feb 3;29(2):464-488. doi: 10.1016/j.ymthe.2020.12.007. Epub 2020 Dec 10. Mol Ther. 2021. PMID: 33309881 Free PMC article. Review.
We review gene therapy for neuromuscular disorders (spinal muscular atrophy [SMA]; Duchenne muscular dystrophy [DMD]; X-linked myotubular myopathy [XLMTM]; and diseases of the central nervous system, including Alzheimer's disease, Parkinson's diseas
We review gene therapy for neuromuscular disorders (spinal muscular atrophy [SMA]; Duchenne muscular dystrophy [DMD]; X-linked myotubular my …
Mycoplasma pneumoniae-induced rash and mucositis as a syndrome distinct from Stevens-Johnson syndrome and erythema multiforme: a systematic review.
Canavan TN, Mathes EF, Frieden I, Shinkai K. Canavan TN, et al. J Am Acad Dermatol. 2015 Feb;72(2):239-45. doi: 10.1016/j.jaad.2014.06.026. J Am Acad Dermatol. 2015. PMID: 25592340 Review.
OBJECTIVE: We sought to review the literature characterizing morphology and disease course of M pneumoniae-associated mucocutaneous disease. METHODS: A comprehensive literature search identified 95 articles with 202 cases. ...Because of the distinct morphology, mild …
OBJECTIVE: We sought to review the literature characterizing morphology and disease course of M pneumoniae-associated mucocutaneous …
Leukodystrophy Imaging: Insights for Diagnostic Dilemmas.
Thakkar RN, Patel D, Kioutchoukova IP, Al-Bahou R, Reddy P, Foster DT, Lucke-Wold B. Thakkar RN, et al. Med Sci (Basel). 2024 Jan 25;12(1):7. doi: 10.3390/medsci12010007. Med Sci (Basel). 2024. PMID: 38390857 Free PMC article. Review.
The leukodystrophies discussed in this paper include X-linked adrenoleukodystrophy, metachromatic leukodystrophy, Krabbe's disease, Pelizaeus-Merzbacher disease, Alexander's disease, Canavan disease, and Aicardi-Goutieres Syndrome....
The leukodystrophies discussed in this paper include X-linked adrenoleukodystrophy, metachromatic leukodystrophy, Krabbe's disease, P …
Leukodystrophies.
Perlman SJ, Mar S. Perlman SJ, et al. Adv Exp Med Biol. 2012;724:154-71. doi: 10.1007/978-1-4614-0653-2_13. Adv Exp Med Biol. 2012. PMID: 22411242 Review.
The clinical characteristics, etiologies, diagnostic testing and treatment options are reviewed in detail for some of the major leukodystrophies: X-linked adrenoleukodystrophy, Krabbe disease, metachromatic leukodystrophy, Pelizaeus-Merzbacher disease, Alexander …
The clinical characteristics, etiologies, diagnostic testing and treatment options are reviewed in detail for some of the major leukodystrop …
Neurosurgical gene therapy for central nervous system diseases.
Patel RV, Nanda P, Richardson RM. Patel RV, et al. Neurotherapeutics. 2024 Jul;21(4):e00434. doi: 10.1016/j.neurot.2024.e00434. Epub 2024 Aug 26. Neurotherapeutics. 2024. PMID: 39191071 Free PMC article. Review.
A range of CNS conditions across pediatric and adult populations are being studied with CNS targeted viral vector gene therapy, including Alzheimer's disease, Parkinson's disease, AADC deficiency, sphingolipidoses, mucopolysaccharidoses, neuronal ceroid lipofuscinos …
A range of CNS conditions across pediatric and adult populations are being studied with CNS targeted viral vector gene therapy, including Al …
Magnetic resonance spectroscopy.
Gujar SK, Maheshwari S, Björkman-Burtscher I, Sundgren PC. Gujar SK, et al. J Neuroophthalmol. 2005 Sep;25(3):217-26. doi: 10.1097/01.wno.0000177307.21081.81. J Neuroophthalmol. 2005. PMID: 16148633 Review.
The most widely used clinical application of MRS has been in the evaluation of central nervous system disorders.MRS has its limitations and is not always specific but, with good technique and in combination with clinical information and conventional MRI, can …
The most widely used clinical application of MRS has been in the evaluation of central nervous system disorders.MRS has …
Gene therapy for the leukodystrophies: From preclinical animal studies to clinical trials.
Metovic J, Li Y, Gong Y, Eichler F. Metovic J, et al. Neurotherapeutics. 2024 Jul;21(4):e00443. doi: 10.1016/j.neurot.2024.e00443. Epub 2024 Sep 13. Neurotherapeutics. 2024. PMID: 39276676 Free PMC article. Review.
Of the approximately 50 leukodystrophies described to date, only eight have existing gene therapy clinical trials: metachromatic leukodystrophy, X-linked adrenoleukodystrophy, globoid cell leukodystrophy, Canavan disease, giant axonal neuropathy, GM2 gangliosidoses, …
Of the approximately 50 leukodystrophies described to date, only eight have existing gene therapy clinical trials: metachromatic leukodystro …
Cervical cancer.
Canavan TP, Doshi NR. Canavan TP, et al. Am Fam Physician. 2000 Mar 1;61(5):1369-76. Am Fam Physician. 2000. PMID: 10735343 Free article. Review.
Accurate and early recognition of abnormal cytologic changes prevents progression of the disease from preinvasive to invasive. Research is under way to determine if efforts to reduce the false-negative rate of the Papanicolaou smear should include rescreening programs and …
Accurate and early recognition of abnormal cytologic changes prevents progression of the disease from preinvasive to invasive. Resear …
Leukodystrophies with astrocytic dysfunction.
Rodriguez D. Rodriguez D. Handb Clin Neurol. 2013;113:1619-28. doi: 10.1016/B978-0-444-59565-2.00030-7. Handb Clin Neurol. 2013. PMID: 23622383 Review.
Astrocytic dysfunctions have been recently identified in four leukosdystrophies without peripheral nervous system myelin involvement. Alexander disease, the first primary genetic astrocytic disorder identified, is due to dominant GFAP mutations. ...An increas …
Astrocytic dysfunctions have been recently identified in four leukosdystrophies without peripheral nervous system myelin invol …
N-acetyl-aspartate metabolism at the interface of cancer, immunity, and neurodegeneration.
Krause N, Wegner A. Krause N, et al. Curr Opin Biotechnol. 2024 Feb;85:103051. doi: 10.1016/j.copbio.2023.103051. Epub 2023 Dec 15. Curr Opin Biotechnol. 2024. PMID: 38103520 Free article. Review.
This review delineates the critical role of NAA across various cell types and its significance in pathophysiological contexts, including Canavan disease and cancer metabolism. Although traditionally linked with myelination and aspartoacylase-driven carbon donation, …
This review delineates the critical role of NAA across various cell types and its significance in pathophysiological contexts, including …
104 results