Gene Therapies for Monogenic Autism Spectrum Disorders
Round 1
Reviewer 1 Report
no comments
Author Response
Dear Reviewer 1, we thank you for reviewing our manuscript and we have succesfully uploaded a new version.
Best,
Wout Weuring,
Also on behalf of
Jeroen Geerligs
Bobby Koeleman
Reviewer 2 Report
This article by Koeleman and colleagues is a well written, comprehensive review on the permanent novel genome editing and transient gene therapies that have been developed in the past 7 years. The results also highlight the journey of developing the first in-human clinical trials for monogenic disorders. Syndromic autism spectrum disorders can be caused by mutations in a single gene. This monogenic aspect and severity of syndromic ASD, makes it an ideal candidate for gene therapies. The review report summarises eleven monogenic ASD syndromes, validated by animal models and reviews current gene therapies for each syndrome.
The contents of this review article lies within the aims and scope of the Journal ‘Genes’, as it highlights the genes potentially being targeted by genomic and non-genome editing strategies for therapeutic intervention of monogenic ASD. The work is of high relevance as it summarises the journey of attempts of various scientists around the world, to find suitable technology of genetic manipulations with an aim and objective to find a cure for ASD.
However, I feel there are few formatting and organization strategies if adopted, will make the manuscript more appealing for readers.
- Firstly Figure 1, can be the schematic consisting of transient and permanent gene therapies.
- Then two separate tables should be generated summarizing models, literature and kind of transient gene therapies used. This table is already present but needs to be separated from the schematic model. A similar table should form the second table of the review, summarising the permanent gene therapies being tested, used or in literature cited , for monogenic autism spectrum disorders.
Author Response
Dear Reviewer 2,
We thank you for your kind words. We have adressed all your points and include a new version of the manuscript.
1) English language and style errors have been reviewed using the Genes' rapid edit service. We kindly thank Miss. Morgan Hekking for her work.
2) We splitted the original Figure 1A /1B to Figure 1 and Table 1, both with captions including abbreviations. Links in the text have been altered
In addition we made minor revisions to some of the paragraphs, and one sentence in the discussion to create a better flow.
