Perspective, Cell Biol Vol: 12 Issue: 3

Cellular Insights into CRISPR-Cas9 in Precision Medicine

Yioz Weing^*

¹Department of Internal Medicine, University of Texas Southwestern Medical Center, Dallas, USA

*Corresponding Author: Yioz Weing,
Department of Internal Medicine, University of Texas Southwestern Medical Center, Dallas, USA
E-mail: weingyioz7@usd.edu

Received date: 02 September, 2023, Manuscript No. CBRT-23-116138;

Editor assigned date: 04 September, 2023, PreQC No CBRT-23-116138 (PQ);

Reviewed date: 18 September, 2023, QC No CBRT-23-116138;

Revised date: 25 September, 2023, Manuscript No CBRT-23-116138 (R);

Published date: 05 October, 2023, DOI: 10.4172/2324-9293.1000186.

Citation: Weing Y (2023) Cellular Insights into CRISPR-Cas9 in Precision Medicine. Cell Biol 12:3.

Description

Precision medicine has revolutionized the field of healthcare by tailoring treatments to individual patients' genetic makeup. Among the various technologies enabling this revolution, the CRISPR-Cas9 gene editing system stands out as a powerful tool for cellular-level precision.

Precision medicine seeks to provide patients with tailored treatments that consider their unique genetic variations, improving efficacy and reducing side effects. The CRISPR-Cas9 system, a breakthrough in gene editing, has become a linchpin in the pursuit of precision medicine by enabling precise genome modifications at the cellular level.

Cellular mechanisms of Crispr-cas9

DNA cleavage and repair: CRISPR-Cas9 uses guide RNA sequences to target specific genes within the genome. Cas9, an endonuclease, which makes Double-Strand Breaks (DSBs) at the target site. Cellular repair mechanisms, such as Non-Homologous End Joining (NHEJ) or Homology-Directed Repair (HDR), are subsequently activated to repair the DSB.

Cellular response to CRISPR-Cas9: Understanding how cells respond to CRISPR-Cas9 is crucial for minimizing off-target effects and optimizing editing efficiency. Cellular factors like p53 and play roles in Deoxyribonucleic Acid (DNA) damage response pathways, influencing the outcome of CRISPR-Cas9 editing.

Cellular insights informing precision medicine: Target Identification: Cell biology techniques are essential for identifying precise targets for CRISPR-Cas9 editing. Single-cell RNA sequencing and proteomics help researchers pinpoint genes and proteins involved in disease pathways. These insights guide the selection of therapeutic targets.

Genetic variations: Cellular studies reveal the diversity of genetic variations among individuals. CRISPR-Cas9 can be adapted to correct or modulate these variations, allowing for personalized therapies. Cellular models, such as patient-derived induced Pluripotent Stem Cells (iPSCs), enable researchers to assess the efficacy of these edits.

Disease modeling: Cellular insights have led to the development of disease-specific cellular models. CRISPR-Cas9 can introduce diseaseassociated mutations into these models to study disease mechanisms and test potential therapies. This approach accelerates drug discovery and development.

Applications in precision medicine

Cancer therapy: Cellular insights into CRISPR-Cas9 have fueled the development of cancer immunotherapies. CAR-T cell therapies, for example, use CRISPR-Cas9 to engineer patient-derived T cells to target cancer-specific antigens, improving the precision and efficacy of treatment.

Inherited genetic disorders: Cellular models derived from patientspecific iPSCs are instrumental in studying and correcting genetic mutations causing disorders like cystic fibrosis and muscular dystrophy. CRISPR-Cas9 holds promise for personalized gene therapy in these conditions.

Neurodegenerative diseases: Cellular insights have enabled the development of cellular models for neurodegenerative diseases like Alzheimer's and Parkinson's. CRISPR-Cas9 is used to introduce disease-related mutations, aiding in drug discovery and potential gene therapies.

Challenges and future directions: While cellular insights have propelled CRISPR-Cas9 to the forefront of precision medicine, challenges remain. Off-target effects, delivery methods, and ethical considerations require ongoing research and innovation. Additionally, expanding the use of CRISPR-Cas9 to more complex diseases and tissues poses exciting yet complex prospects.

Conclusion

Cellular insights into CRISPR-Cas9 have catalyzed advancements in precision medicine. By understanding the cellular mechanisms and responses to gene editing, researchers are optimizing this technology for therapeutic applications. CRISPR-Cas9's potential to target specific genes at the cellular level is ushering in a new era of personalized medicine, offering hope to patients with a wide range of diseases. As it continues to unlock cellular insights, the path to precision medicine becomes increasingly promising.