Keyword: Precision Medicine
2 results found.
Review Article
Australian Journal of Biomedical Research, 2(3), 2026, aubm024, https://doi.org/10.63946/aubiomed/18967
ABSTRACT:
Background: CRISPR-Cas9 has revolutionized genome editing by providing an efficient, versatile, and comparatively accessible platform for targeted genetic modification. Its rapid progression from laboratory innovation to clinical application has generated substantial interest across multiple therapeutic domains, while simultaneously raising significant safety, ethical, and regulatory concerns.
Objective: This scoping review aimed to comprehensively map current evidence regarding the therapeutic applications, safety challenges, and ethical frameworks associated with CRISPR-Cas9 in human health.
Methods: A scoping review methodology guided by the Arksey and O’Malley framework and PRISMA-ScR guidelines was employed. Comprehensive searches were conducted across PubMed, Dimensions, and Embase for peer-reviewed English-language studies published between 2015 and 2026. Eligible studies focused on CRISPR-Cas9 applications in human health, including therapeutic interventions, safety evaluations, ethical analyses, and regulatory considerations. A total of 32 studies met the inclusion criteria and underwent thematic synthesis.
Results: CRISPR-Cas9 demonstrated substantial therapeutic promise across hematologic disorders, metabolic diseases, ophthalmologic conditions, oncology, immunotherapy, and rare genetic disorders. The most clinically advanced applications were observed in sickle cell disease, β-thalassemia, hereditary transthyretin amyloidosis, and hereditary angioedema, where clinical trials showed durable and potentially curative outcomes. However, major barriers remain, including off-target effects, genomic instability, delivery inefficiencies, immunogenicity, and limited long-term safety data. Ethical and regulatory concerns were prominent, particularly regarding germline editing, health equity, global governance, and accessibility.
Conclusion: CRISPR-Cas9 has demonstrated considerable clinical potential across a range of therapeutic applications, particularly for selected monogenic disorders in which the strongest clinical evidence is currently available. Although important advances have been achieved, broader clinical implementation will require continued improvements in editing precision, long-term safety evaluation, delivery technologies, ethical oversight, equitable access, and harmonized regulatory frameworks.
Objective: This scoping review aimed to comprehensively map current evidence regarding the therapeutic applications, safety challenges, and ethical frameworks associated with CRISPR-Cas9 in human health.
Methods: A scoping review methodology guided by the Arksey and O’Malley framework and PRISMA-ScR guidelines was employed. Comprehensive searches were conducted across PubMed, Dimensions, and Embase for peer-reviewed English-language studies published between 2015 and 2026. Eligible studies focused on CRISPR-Cas9 applications in human health, including therapeutic interventions, safety evaluations, ethical analyses, and regulatory considerations. A total of 32 studies met the inclusion criteria and underwent thematic synthesis.
Results: CRISPR-Cas9 demonstrated substantial therapeutic promise across hematologic disorders, metabolic diseases, ophthalmologic conditions, oncology, immunotherapy, and rare genetic disorders. The most clinically advanced applications were observed in sickle cell disease, β-thalassemia, hereditary transthyretin amyloidosis, and hereditary angioedema, where clinical trials showed durable and potentially curative outcomes. However, major barriers remain, including off-target effects, genomic instability, delivery inefficiencies, immunogenicity, and limited long-term safety data. Ethical and regulatory concerns were prominent, particularly regarding germline editing, health equity, global governance, and accessibility.
Conclusion: CRISPR-Cas9 has demonstrated considerable clinical potential across a range of therapeutic applications, particularly for selected monogenic disorders in which the strongest clinical evidence is currently available. Although important advances have been achieved, broader clinical implementation will require continued improvements in editing precision, long-term safety evaluation, delivery technologies, ethical oversight, equitable access, and harmonized regulatory frameworks.
Review Article
Australian Journal of Biomedical Research, 1(1), 2025, aubm001, https://doi.org/10.63946/aubiomed/16743
ABSTRACT:
Background: Personalised medicine through pharmacogenomics is revolutionalizing healthcare delivery by encouraging individualized therapy that takes into consideration an individual's genetic profile, environment and lifestyle. Pharmacogenomics is an aspect of pharmacy that studies the relationship between genetic profile and response to therapeutic agents. However, the application of the concepts of pharmacogenomics in healthcare helps in achieving more effective and safe responses from therapy. This study evaluates the application and benefits of pharmacogenomics in clinical practice based on evidence from current practices in various medical fields.
Methods: In carrying out this review, PubMed database was the primary literature source and we analyzed and synthesized findings from the included literature thematically as it relates to pharmacogenomics applications, benefits and challenges as well as safety and ethical concerns.
Results: Pharmacogenomics has been widely applied in various aspects of healthcare such as in dosing, choice of treatment, reducing and management of adverse reactions, individualization of therapy, optimizing efficacy of therapy. Despite its numerous applications, its adoption faces challenges such as limited clinical evidence, lack of specialized training among healthcare professionals, cost and complexity of genetic mapping as well as ethical concerns.
Conclusion: With ongoing advances in genomic technologies, pharmacogenomics is becoming an integral aspect of individualization therapy in clinical practice and more widely applied in different healthcare sectors.
Methods: In carrying out this review, PubMed database was the primary literature source and we analyzed and synthesized findings from the included literature thematically as it relates to pharmacogenomics applications, benefits and challenges as well as safety and ethical concerns.
Results: Pharmacogenomics has been widely applied in various aspects of healthcare such as in dosing, choice of treatment, reducing and management of adverse reactions, individualization of therapy, optimizing efficacy of therapy. Despite its numerous applications, its adoption faces challenges such as limited clinical evidence, lack of specialized training among healthcare professionals, cost and complexity of genetic mapping as well as ethical concerns.
Conclusion: With ongoing advances in genomic technologies, pharmacogenomics is becoming an integral aspect of individualization therapy in clinical practice and more widely applied in different healthcare sectors.