Nael Ismail: Gene and Cell Therapy Are Transforming the Treatment of Human Genetic Diseases
Nael Ismail, Clinical Advisor at Vgenomics, shared a post on Linkedin about a recent article by Busra Cetin et al. published in Journal of Cellular and Molecular Medicine, adding:
“Genetic diagnosis should not be the end of the story.
For decades, medical genetics has focused heavily on identifying the variant, establishing the diagnosis, and explaining the disease.
But what happens next?
The therapeutic landscape is changing rapidly.
Gene therapy, cell therapy and CRISPR-based genome editing are moving genetic medicine from describing disease to potentially modifying its molecular cause.
We are already seeing this transition in real patients: spinal muscular atrophy, inherited retinal disease, hemophilia, B-thalassemia, sickle cell disease and cancers are among the areas where advanced genetic and cellular therapies have reached clinical practice.
And yet, our healthcare systems often remain structured around an older model:
Diagnose, then report, refer, and manage symptoms.
That model is increasingly inadequate for some genetic diseases.
The real challenge is no longer simply whether we can identify the molecular abnormality.
It is whether we can translate that molecular knowledge into meaningful clinical action.
Of course, gene and cell therapies come with enormous challenges: delivery, immune responses, off-target effects, durability, manufacturing, long-term safety, regulation and cost.
But these challenges should push us toward better clinical integration, not toward treating genomics as an endpoint.
The future of medical genetics cannot be limited to finding variants.
It must increasingly connect genomic information to molecular mechanisms, therapeutic targets, clinical decisions, and patient outcomes.
That is where medical genetics, clinical genomics, molecular medicine and precision medicine truly converge.
The question is no longer only: ‘What genetic disease does this patient have?’
It is increasingly:
‘Now that we know the molecular cause, what can we actually do about it?'”
Title: Gene and cell therapy of human genetic diseases: Recent advances and future directions
Authors: Busra Cetin, Fulya Erendor, Yunus E Eksi, Ahter D Sanlioglu, Salih Sanlioglu
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