Chromosome therapy: Potential strategies for the correction of severe chromosome aberrations

Area of Science:

• Genetics and Molecular Biology
• Developmental Biology
• Genomic Medicine

Background:

• Large chromosomal aberrations are frequent during development, leading to complex multisystem diseases.
• Current treatments cannot correct these disorders due to their complexity and involvement of multiple genes.
• Recent advancements have introduced novel in vitro approaches targeting entire chromosomes, termed Chromosome Therapies.

Purpose of the Study:

• To review the emerging field of Chromosome Therapy.
• To discuss the clinical potential and research applications of these novel technologies.
• To explore the use of Chromosome Therapies in creating cellular models.

Main Methods:

• Review of existing literature on Chromosome Therapy approaches.
• Discussion of techniques such as silencing and selection for chromosome loss in trisomies.
• Exploration of methods for promoting euploidy in aneuploid cultures and forced chromosome loss/replacement.

Main Results:

• Chromosome Therapies represent a significant advancement in addressing chromosomal abnormalities.
• These therapies offer potential for correcting genetic disorders previously considered untreatable.
• The review highlights the versatility of these methods for both clinical and research purposes.

Conclusions:

• Chromosome Therapies hold promise for future clinical interventions in genetic disorders.
• Further research and development are needed to translate these in vitro techniques into effective treatments.
• These technologies open new avenues for understanding and modeling chromosomal diseases.