Current and Future Advances in Using Molecular Replacement to Treat Diseases

Replacement therapy aims to restore normal physiology by replacing deficient molecules.

• Different modalities include protein replacement, gene therapy, mRNA replacement, noncoding RNA therapies, cell replacement, and gene editing.
• These therapies could address fundamental genetic defects underlying a range of diseases.
• Replacement therapies may offer potentially curative and disease-modifying effects compared to conventional treatments.
• Some therapies, like protein and gene therapy, are already approved for clinical use, while others are still in preclinical or trial stages.
• Key challenges for emerging therapies include delivery, safety, specificity, and ethical concerns.

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