ReviewHuman molecular genetics2011
Genetic therapy for the nervous system.
Review in Human molecular genetics, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 35 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
35 citing papers in PubMed, 71 citations in OpenAlex.
- Review
- Intracranial Gene Delivery Mediated by Albumin-Based Nanobubbles and Low-Frequency Ultrasound.Nanomaterials (Basel, Switzerland) · 2024Article
- DREADDs in Epilepsy Research: Network-Based Review.Frontiers in molecular neuroscience · 2022Review
- Neurospheres: a potential in vitro model for the study of central nervous system disorders.Molecular biology reports · 2021Review
- High rate of HDR in gene editing of p.(Thr158Met) MECP2 mutational hotspot.European journal of human genetics : EJHG · 2020Article
- Non-adhesive and highly stable biodegradable nanoparticles that provide widespread and safe transgene expression in orthotopic brain tumors.Drug delivery and translational research · 2020Article
- Challenges of gene delivery to the central nervous system and the growing use of biomaterial vectors.Brain research bulletin · 2019Review
- On DNA Signatures, Their Dual-Use Potential for GMO Counterfeiting, and a Cyber-Based Security Solution.Frontiers in bioengineering and biotechnology · 2019Article
- Adeno-associated virus 2/9 delivery of Cre recombinase in mouse primary afferents.Scientific reports · 2018Article
- RNA Dysregulation in Amyotrophic Lateral Sclerosis.Frontiers in genetics · 2018Review
- Viral vectors for therapy of neurologic diseases.Neuropharmacology · 2017Review
- Current status of non-viral gene therapy for CNS disorders.Expert opinion on drug delivery · 2016Review
- Intracranial AAV-sTRAIL combined with lanatoside C prolongs survival in an orthotopic xenograft mouse model of invasive glioblastoma.Molecular oncology · 2016Article
- Article
- Gene Therapy Corrects Mitochondrial Dysfunction in Hematopoietic Progenitor Cells and Fibroblasts from Coq9R239X Mice.PloS one · 2016Article
- Engineering humoral immunity as prophylaxis or therapy.Current opinion in immunology · 2015Review
- Neurogenethics: An emerging discipline at the intersection of ethics, neuroscience, and genomics.Applied & translational genomics · 2015Review
- Using magnetic nanoparticles for gene transfer to neural stem cells: stem cell propagation method influences outcomes.Journal of functional biomaterials · 2015Article
- Aging and brain rejuvenation as systemic events.Journal of neurochemistry · 2015Review
- Stages and conformations of the Tau repeat domain during aggregation and its effect on neuronal toxicity.The Journal of biological chemistry · 2014Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors at 3 institutions in 1 country.
Funding
Abstract
Genetic therapy is undergoing a renaissance with expansion of viral and synthetic vectors, use of oligonucleotides (RNA and DNA) and sequence-targeted regulatory molecules, as well as genetically modified cells, including induced pluripotent stem cells from the patients themselves. Several clinical trials for neurologic syndromes appear quite promising. This review covers genetic strategies to ameliorate neurologic syndromes of different etiologies, including lysosomal storage diseases, Alzheimer's disease and other amyloidopathies, Parkinson's disease, spinal muscular atrophy, amyotrophic lateral sclerosis and brain tumors. This field has been propelled by genetic technologies, including identifying disease genes and disruptive mutations, design of genomic interacting elements to regulate transcription and splicing of specific precursor mRNAs and use of novel non-coding regulatory RNAs. These versatile new tools for manipulation of genetic elements provide the ability to tailor the mode of genetic intervention to specific aspects of a disease state.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.