Question-2

Last updated: 2022-07-11

Checks: 7 0

Knit directory: prelim/

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R Markdown file: up-to-date

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Environment: empty

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Seed: set.seed(20220706)

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Session information: recorded

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Cache: none

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File paths: relative

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Repository version: 80ddacb

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The results in this page were generated with repository version 80ddacb. See the Past versions tab to see a history of the changes made to the R Markdown and HTML files.

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Untracked files:
    Untracked:  notes/other/

Unstaged changes:
    Deleted:    notes/questions_not_selected.Rmd

Note that any generated files, e.g. HTML, png, CSS, etc., are not included in this status report because it is ok for generated content to have uncommitted changes.

These are the previous versions of the repository in which changes were made to the R Markdown (notes/question_2.Rmd) and HTML (docs/question_2.html) files. If you’ve configured a remote Git repository (see ?wflow_git_remote), click on the hyperlinks in the table below to view the files as they were in that past version.

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Materials

Question-2 (by Bruce Lahn)
Q2 refs: Ref-1, Ref-2, Ref-3

Questions

As a concept, gene therapy has been around for decades. But it has only recently emerged as an effective therapeutic modality with a number of drugs getting approved in the last few years. With gene therapy finally moving beyond the proof-of-concept stage, the field is poised to flourish, with many previously incurable diseases now expected to have complete cures in the coming years.

Please describe the major classes of diseases targeted by gene therapy either as approved treatments or at the research stage. Please explain why these diseases are particularly conducive to gene therapy. Conversely, please describe what types of diseases are not conducive to gene therapy at present and why.
What are the major limitations and challenges in the effective use of gene therapy?
Lentivirus (LV) and adeno-associated virus (AAV) are currently the most popular gene therapy vectors. Please describe the pros and cons of LV and AAV, and what types of diseases they are each suitable for.

Answers

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sessionInfo()

R version 4.2.1 (2022-06-23)
Platform: x86_64-pc-linux-gnu (64-bit)
Running under: Ubuntu 22.04 LTS

Matrix products: default
BLAS:   /usr/lib/x86_64-linux-gnu/blas/libblas.so.3.10.0
LAPACK: /usr/lib/x86_64-linux-gnu/lapack/liblapack.so.3.10.0

locale:
 [1] LC_CTYPE=en_US.UTF-8       LC_NUMERIC=C              
 [3] LC_TIME=en_US.UTF-8        LC_COLLATE=en_US.UTF-8    
 [5] LC_MONETARY=en_US.UTF-8    LC_MESSAGES=en_US.UTF-8   
 [7] LC_PAPER=en_US.UTF-8       LC_NAME=C                 
 [9] LC_ADDRESS=C               LC_TELEPHONE=C            
[11] LC_MEASUREMENT=en_US.UTF-8 LC_IDENTIFICATION=C       

attached base packages:
[1] stats     graphics  grDevices utils     datasets  methods   base     

loaded via a namespace (and not attached):
 [1] Rcpp_1.0.8.3     rstudioapi_0.13  whisker_0.4      knitr_1.39      
 [5] magrittr_2.0.3   workflowr_1.7.0  R6_2.5.1         rlang_1.0.2     
 [9] fastmap_1.1.0    fansi_1.0.3      stringr_1.4.0    tools_4.2.1     
[13] xfun_0.31        utf8_1.2.2       cli_3.3.0        git2r_0.30.1    
[17] jquerylib_0.1.4  htmltools_0.5.2  ellipsis_0.3.2   rprojroot_2.0.3 
[21] yaml_2.3.5       digest_0.6.29    tibble_3.1.7     lifecycle_1.0.1 
[25] crayon_1.5.1     later_1.3.0      sass_0.4.1       vctrs_0.4.1     
[29] fs_1.5.2         promises_1.2.0.1 glue_1.6.2       evaluate_0.15   
[33] rmarkdown_2.14   stringi_1.7.6    bslib_0.3.1      compiler_4.2.1  
[37] pillar_1.7.0     jsonlite_1.8.0   httpuv_1.6.5     pkgconfig_2.0.3