CRISPR/Cas9 Genome Editing for Tissue-Specific In Vivo Targeting: Nanomaterials and Translational Perspective

Sahel DK, Vora LK, Saraswat A, et al. CRISPR/Cas9 Genome Editing for Tissue-Specific In Vivo Targeting: Nanomaterials and Translational Perspective. Advanced Science (Weinheim, Baden-wurttemberg, Germany). 2023 Jul;10(19):e2207512. DOI: 10.1002/advs.202207512. PMID: 37166046; PMCID: PMC10323670. 

Despite DSBs, they have also been explored through epigenome editing and biosensor applications. However, its in vivo delivery is still a major challenge. Interestingly, ample nonviral nanocarriers have been developed and explored for the in vivo delivery of CRISPR components, but they are limited to some immune-prone organs of the body, such as the liver, lungs, or spleen. 

However, various pharmaceutical companies are showing interest in the CRISPR therapeutics area, and therefore, it will be interesting to watch the progress in this field. Since both ethical and social implications are associated with the usage of CRISPR, we need to move forward very responsibly.

Preparing for Disease-Modification Trials in Degenerative Cerebellar Ataxias: Which Endpoints to Choose?

Maas RPPWM. Preparing for Disease-Modification Trials in Degenerative Cerebellar Ataxias: Which Endpoints to Choose? Movement Disorders : Official Journal of the Movement Disorder Society. 2023 Jun;38(6):917-923. DOI: 10.1002/mds.29388. PMID: 37475615.

Brain MRI detects early-stage alterations and disease progression in Friedreich ataxia

Adanyeguh IM, Joers JM, Deelchand DK, et al. Brain MRI detects early-stage alterations and disease progression in Friedreich ataxia. Brain Communications. 2023 ;5(4):fcad196. DOI: 10.1093/braincomms/fcad196. PMID: 37483529. 

 We show that fixel-based analysis of diffusion MRI data is particularly sensitive to longitudinal change in the cerebellar peduncles, as well as motor and sensory white matter tracts. In combination with other morphometric measures, they may therefore provide sensitive imaging biomarkers of disease progression for clinical trials.