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Papers, explained in our own words

Every entry summarises what the study set out to test, what it found and why it changes how we design delivery systems. Browse research and reviews or search the collection.

706 articles

Showing 1–15 of 15 articles · Keyword: Gene Therapy Clear keyword

Current cardiology reports2026ReviewNon-viral Gene Delivery

1. Viral Gene Therapy for Danon Disease: From LAMP2 Biology to Clinical Translation

Gunes Ea, Patel U, Iyer S, Taub A, Lee-Guest E, Cline S, Et Al.

Danon disease is a rare, highly penetrant and morbid X-linked cardioskeletal myopathy caused by loss-of-function variants in the lysosome-associated membrane protein 2 (LAMP2) gene. We review the rationale for LAMP2B gene replacement, the basis for adeno-associated virus serotype 9 (AAV9) vector selection, the…

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Annals of allergy, asthma & immunology : official publication of the American College of Allergy, Asthma, & Immunology2026ReviewNon-viral Gene Delivery

2. CRISPR, mRNA, and Beyond: Nucleic Acid Therapeutics in Allergy and Immunology

Schauberger Em, Phillips Ja

The management of allergic and immunologic diseases has long centered on symptom management rather than disease modification. Conventional pharmacotherapy controls manifestations of the immune dysregulation but does little to treat the underlying cause. Newer monoclonal and small molecule therapies target downstream…

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MedComm2026ReviewNon-viral Gene DeliveryDrug Delivery

3. CRISPR Technology: From Bench to Bedside Applications

Ma X, Li L, Sun Y

CRISPR-Cas genome editing represents one of the most transformative advances in modern molecular biology, enabling programmable site-specific genetic modification that has revolutionized functional genomics and precision therapeutic development. Beyond canonical Cas9 nucleases, the CRISPR toolkit has diversified into…

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Materials today. Bio2026ReviewNon-viral Gene Delivery

4. Progress and applications of microneedle-based gene therapy

Shen Y, Ye X, Chen Q, Yu D, He L, Lei L, Et Al.

Gene therapy holds considerable promise for treating a wide range of intractable diseases through the modification of specific genes. However, gene therapeutics such as plasmid DNA, messenger RNA, and CRISPR-based systems are susceptible to degradation, face challenges in crossing cellular barriers, and may induce…

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Molecular medicine reports2026ReviewNon-viral Gene Delivery

5. Gene therapy for epilepsy: From SCN1A to broadened therapeutic horizons (Review)

Qin R, Hu X, Jiang W, Wang J, Zheng M, Zhang Z, Et Al.

Genetic epilepsy, for which the molecular mechanisms are relatively well defined in numerous monogenic forms, represents an attractive model for the transition from symptomatic seizure control to mechanism‑driven correction. However, gene therapy strategies differ in their mechanisms of action, adaptability to…

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Nanomedicine (London, England)2026ReviewNon-viral Gene Delivery

6. Dual-vector nanoengineered mRNA-CRISPR delivery systems for precision gene therapy in choroidal melanoma

Thakur Sk, Malviya R, Chandra P, Sridhar Sb, Shareef J, Wadhwa T

Choroidal melanoma is the most common primary intraocular malignancy, and effective gene therapy remains limited by ocular barriers, tumor heterogeneity, and inefficient nucleic acid delivery. This review critically analyses the molecular rationale, engineering principles, emerging nanoarchitectures, biological…

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Journal of orthopaedic translation2026ReviewNon-viral Gene DeliveryDrug Delivery

7. Nanoparticle delivery systems for gene therapy of degenerative musculoskeletal diseases

Chen X, Sun J, Xie W, Arthur Vithran Dt, Zhang Q, Rosenholm Jm, Et Al.

Degenerative musculoskeletal diseases (DMDs) are a group of chronic conditions characterized by the progressive deterioration of tissue structure and function, primarily affecting the articular cartilage, bones, tendons, and ligaments. With global aging populations, the rising prevalence of these disorders presents a…

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Molecular therapy. Advances2026ReviewNon-viral Gene Delivery

8. Stealthy lentiviral vectors: Breaking the barriers to systemic in vivo gene delivery

Keating Al, Carlisle Rc, Mitrophanous Ka, Seymour Lw, Fisher Kd

Lentiviral vector (LVV)-mediated chimeric antigen receptor (CAR)-T cell therapy has transformed the landscape for blood cancer treatment through genetic modification of patients' T cells. Yet, the complexity of the manufacturing process makes this therapy inaccessible for many patients. Systemic intravenous delivery…

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Molecular therapy. Oncology2026ReviewNon-viral Gene DeliveryDrug Delivery

9. Gene therapy as an upcoming technique in the treatment of cancer: A cellular solution to a cellular problem

Bedada At, Genet S

Cancer remains a formidable global health challenge, representing the second leading cause of mortality worldwide. In 2023, there were approximately 18.5 million new cancer cases and over 10 million deaths globally, with projections estimating 30.5 million new cases and 18.6 million deaths annually by 2050. In the…

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Viruses2026ReviewNon-viral Gene Delivery

10. Gene Therapy for Osteoarthritis: Viral and Non-Viral Delivery Strategies

Zhang M, Mohess J, Cheung J, Du Q, Qiu J, Wang J

Osteoarthritis (OA) is the most prevalent joint disease, which is pathologically characterized by articular cartilage degradation, chronic synovial inflammation, subchondral bone remodeling, and osteophyte formation. Patients with OA suffer from joint pain, deformity, and functional impairment. Current therapies…

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Discover genetics and evolution2026ReviewNon-viral Gene Delivery

11. Navigating off-target effects in CRISPR-based genome editing for safer gene therapies

Bhardwaj A

Off-target activity remains a significant challenge in the clinical development of CRISPR-based therapies. As programmable nucleases advance from experimental tools toward approved medicines, the ability to predict, detect, and mitigate unintended genomic editing events has acquired direct translational importance.

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Advances in Colloid and Interface Science2026ReviewNon-viral Gene DeliveryDrug Delivery

12. Advances in lipids design for LNP-mediated DNA and RNA delivery

D Zhi, A Halemaimaiti, B Dai, H Li, H Wang, Z Zhang, J Yu, S Zhang

This review examines how lipid structure and formulation affect the delivery of DNA, mRNA and siRNA by lipid nanoparticles, while assessing tissue targeting, safety and clinical translation challenges.

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BioDrugs2026ReviewNon-viral Gene DeliveryDrug Delivery

13. Recent Developments in Lipid Nanoparticle-Mediated Delivery of Biotherapeutics and Gene Therapy Across the Blood-Brain Barrier

U Iqbal, Rw Hwang, Wj Costain

A review of blood–brain barrier transport mechanisms and emerging lipid nanoparticle strategies for carrying nucleic acid and other complex therapies into the central nervous system.

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