ArticleMolecular therapy. Methods & clinical development2020
Optimization of
Article in Molecular therapy. Methods & clinical development, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 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
11 citing papers in PubMed.
- CRISPR Interference to Inhibit Oncogenes for Cancer Therapy.International journal of molecular sciences · 2026Review
- Sensory-neuron-derived CGRPα controls white adipocyte differentiation and tissue plasticity.Cell reports · 2025Article
- The human glucocorticoid receptor variant rs6190 increases blood cholesterol and promotes atherosclerosis.The Journal of clinical investigation · 2025Article
- Nanoscale synthetic biology with innovative medicinal applications.Fundamental research · 2025Review
- CRISPR-based functional genomics tools in vertebrate models.Experimental & molecular medicine · 2025Review
- Discovery of Diverse CRISPR-Cas Systems and Expansion of the Genome Engineering Toolbox.Biochemistry · 2023Review
- NRF2/ARE mediated antioxidant response to glaucoma: role of glia and retinal ganglion cells.Acta neuropathologica communications · 2023Article
- Advances in Recombinant Adeno-Associated Virus Vectors for Neurodegenerative Diseases.Biomedicines · 2023Review
- Toward the Development of Epigenome Editing-Based Therapeutics: Potentials and Challenges.International journal of molecular sciences · 2023Review
- CRISPR/Cas9 therapeutics: progress and prospects.Signal transduction and targeted therapy · 2023Review
- Delivery Systems of Retinoprotective Proteins in the Retina.International journal of molecular sciences · 2021Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors.
Funding
Abstract
The power of CRISPRi to decrease targeted gene expression for clinical applications has been inhibited by delivery challenges. Existing constructs are too large to fit within the ∼4.7 kb packaging size limitation of adeno-associated virus (AAV), the only FDA approved viral vector for clinical use. Therefore, we optimized CRISPRi components to generate a single AAV vector that contains all functional elements and effectively knocks down expression of an endogenous gene
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.