Question

from the evidence figure i gave you is there any figure that shows crispr fixing single gene disorder

The median time period since survey respondents had first investigated or started using CRISPR/Cas 9 gene editing technology was 6-12 months ago (Figure 2).Most (47%) of survey respondents were applying or intending to apply CRISPR/Cas 9 gene editing to the basic research area of the drug discovery process. This was followed by target validation (18% applying); target identification (10% applying) and then clinical studies and lead generation (both with 9% applying).

Figure 2 Time period since first started using CRISPR/Cas9 gene editing technology
Other drug discovery areas had in total only 6% applying (Figure 3).

Figure 3 Area of the drug discovery process most applying or intending to apply CRISPR/Cas9 gene editing
Oncology/cancer was the key disease or research area most targeted by survey respondents (52% targeting) by CRISPR/Cas 9 gene editing. This was followed by immunology/inflammatory disease/ autoimmune (30% targeting); neurology/CNS/ neurodegeneration/pain (27% targeting); metabolic disease/diabetes (18% targeting); and then cardiovascular disease (17% targeting) (Figure 4).

Figure 4 Key diseases or research areas using CRISPR/Cas9 gene editing
Most (49%) of survey respondents answered N/A, ie they have not investigated other gene editing technologies prior to CRISPR/Cas 9 availability. 29% have previously investigated transcription activator-like effector nucleases (TALENs); 21% have investigated integration via homologous recombination (eg with rAAV); 16% zinc finger nucleases (ZFN); and 11% other approaches (Figure 5).

Figure 5 Other gene editing technologies investigated prior to CRISPR/Cas9
Main rationale for using CRISPR/Cas9 gene editing technology
A gene knockout was what the majority (77%) of survey respondents most want to achieve using CRISPR/Cas 9 gene editing technology. This was followed by introduce defined mutations, insertions or modifications (62% wanting); gene knock-in (52% wanting); and then gene knock down (inducible) (40% wanting). Other aims were wanted by less than a third of survey respondents (Figure 6).

Figure 6 What respondents want to achieve using CRISPR/Cas9 gene editing
Identification of new therapeutic targets was the main objective of survey respondents CRISPR/Cas 9 gene editing in drug discovery (61% wanting). This was followed by validation of new therapeutic targets (48% wanting); and then investigation of mechanism of action and screens to identify genes that regulate various cell biological processes (both with 46% wanting). Of least interest was deconvolution and validation of GWAS hits (only 8% targeting) (Figure 7).

Figure 7 Main objectives of CRISPR/Cas9 gene editing in drug discovery
Survey respondents ranked some of the perceived advantages of CRISPR/Cas 9 gene editing technology in order of importance. This analysis revealed that efficient, ie edit targets sequences at surprisingly high rates, was seen as the main advantage of CRISPR/Cas 9 gene editing technology. This was followed by simplicity, ie easy to use and design; programmable, ie get precision targeting; and then fast, ie get tangible results within weeks. Ranked least advantageous was multiplexing, ie can programme multiple guide RNAs and cleave multiple genes simultaneously (Figure 8).

Figure 8 The advantages of CRISPR/Cas9 gene editing technology
Survey respondents rated complete genetic knockout, while minimising off-target effects as the potential benefit of CRISPR/Cas 9 in drug discovery they were most interested in exploiting. This was closely followed by enables rapid generation of cell lines harbouring desired mutations; develop accurate models of complex human disease in an efficient manner; and then relative ease with which cellular models can be generated. Rated least interesting was scalable generation of genome-wide CRISPR libraries for HT functional genomics screening (Figure 9).

Figure 9 Potential benefits of CRISPR/Cas9 in drug discovery most respondents want to exploit
Validating gene edits
The downstream analytical technique most used to validate CRISPR/Cas 9 gene edits was PCR (70% using). This was followed by real time PCR (59% using); Western blotting (55% using); FACS (34% using) and then next gen sequencing (miSEQ) (30% using). Least used were capillary electrophoresis, TIDE assay and other DNA mismatch assays (Figure 10).

Figure 10 Downstream analytical techniques used to validate CRISPR/Cas9 gene editing
Multiplexing gene edits
For each single target gene survey respondents have attempted to edit they have designed a median of two single guide RNAs (ie 2 sgRNAs). Interest in simultaneously delivering sgRNAs targeting multiple genes to multiplex CRISPR/Cas 9 gene edits is presented in Figure 11. This showed that the majority (69%) of survey respondents are not yet undertaking multiplexing, but plan future investigation. This was followed by 25% not undertaking with no plans to investigate, leaving only 5% actually undertaking multiplexing today (Figure 11).

Answer 1

[AI-generated answer — Bot GPT 3.5]

Based on the evidence provided, there is no specific figure or data that shows CRISPR fixing single gene disorders. The focus of the survey respondents using CRISPR/Cas9 gene editing technology seems to be more on applications in drug discovery, targeting various diseases and research areas, and achieving specific genetic modifications such as gene knockouts, insertions, or modifications. The main objectives mentioned include identifying new therapeutic targets and validating them, as well as investigating mechanisms of action and screening genes that regulate cell processes. Additionally, the survey respondents highlighted the advantages and potential benefits of CRISPR/Cas9 technology in drug discovery. However, there is no direct mention of fixing single gene disorders in the provided figures.