Targeted Gene Therapy: Promises and Challenges in Disease Management DOI Creative Commons

A.S. Vickram,

Sivasubaramanian Manikandan,

Titus Richard

и другие.

Journal of Bio-X Research, Год журнала: 2024, Номер 7

Опубликована: Янв. 1, 2024

Targeted gene therapy may revolutionize disease treatment by precisely treating genetic defects. This method targets particular cells or tissues with therapeutic genes to treat a variety of problems, including cancer, hereditary diseases, and viral infections. Viral, nonviral, genome editing techniques such as CRISPR-Cas9 are used for targeted fix modify disease-causing minimal off-target effects. The issues vector immunogenicity, mutations, delivery target persist despite tremendous progress. successful implementation is further hindered illness complexity background diversity. These difficulties require multidisciplinary cooperation, novel design, thorough preclinical clinical assessments. long-term unforeseen effects must also be considered from an ethical viewpoint. has considerable promise, but more research technological advances needed overcome limitations develop safe treatments.

Язык: Английский

Synthetic Gene Circuits as a Promising Approach in Cancer Immunotherapy DOI

Bhanuprakash Ananthakumar,

Nagashri Nanjundeshwara,

Archana Lakshmaiah

и другие.

Advances in medical diagnosis, treatment, and care (AMDTC) book series, Год журнала: 2024, Номер unknown, С. 391 - 418

Опубликована: Авг. 28, 2024

Cancer immunotherapy has emerged as a revolutionary approach in the fight against cancer. Unlike traditional treatments like chemotherapy and radiation, harnesses power of body's own immune system to identify destroy cancer cells. promising therapy, but limitations specificity control hinder its full potential. Synthetic gene circuits offer address these challenges. This chapter emphasizes diverse applications synthetic immunotherapy. Additionally, authors discuss advantages AND gate for minimizing off-target effects, engineered bacteria targeted tumour manipulation, T-cell engineering enhanced anti-tumour activity. Ultimately, therapies are not mutually exclusive. While proven effectiveness accessibility, hold immense promise personalized, long-term solutions.

Язык: Английский

Процитировано

5

Extracellular Peptide-Ligand Dimerization Actuator Receptor Design for Reversible and Spatially Dosed 3D Cell-Material Communication DOI Creative Commons
Matthias Recktenwald,

Ritankar Bhattacharya,

Mohammed Mehdi Benmassaoud

и другие.

ACS Synthetic Biology, Год журнала: 2024, Номер unknown

Опубликована: Дек. 20, 2024

Transmembrane receptors that endow mammalian cells with the ability to sense and respond biomaterial-bound ligands will prove instrumental in bridging fields of synthetic biology biomaterials. Materials formed thiol-norbornene chemistry are amenable thiol-peptide patterning, this study reports rational design reversibly activate cellular responses based on peptide-ligand recognition. This transmembrane receptor platform, termed Extracellular Peptide-ligand Dimerization Actuator (EPDA), consists stimulatory or inhibitory pairs come together upon extracellular peptide dimer binding corresponding monobody receptors. Intracellularly, Stimulatory EPDAs phosphorylate a substrate merges two protein halves, whereas Inhibitory revert split proteins back their unmerged, inactive state via dephosphorylation. To identify ligand-receptor pairs, over 2000 candidate monobodies were built silico using PETEI, novel computational algorithm we developed. The top 30 predicted affinity tested experimentally, induced highest change merging (green fluorescent protein, GFP) incorporated final EPDA design. In soluble form, peptides induce intracellular GFP time- concentration-dependent manner, varying levels green fluorescence observed dosing. EPDA-programmed encapsulated hydrogels patterned domains exhibited 3D activation deactivation location within peptide-patterned hydrogels. can recognize myriad peptide-ligands bound materials, beyond fluorescence, widely applicable biological research regenerative medicine.

Язык: Английский

Процитировано

0

Targeted Gene Therapy: Promises and Challenges in Disease Management DOI Creative Commons

A.S. Vickram,

Sivasubaramanian Manikandan,

Titus Richard

и другие.

Journal of Bio-X Research, Год журнала: 2024, Номер 7

Опубликована: Янв. 1, 2024

Targeted gene therapy may revolutionize disease treatment by precisely treating genetic defects. This method targets particular cells or tissues with therapeutic genes to treat a variety of problems, including cancer, hereditary diseases, and viral infections. Viral, nonviral, genome editing techniques such as CRISPR-Cas9 are used for targeted fix modify disease-causing minimal off-target effects. The issues vector immunogenicity, mutations, delivery target persist despite tremendous progress. successful implementation is further hindered illness complexity background diversity. These difficulties require multidisciplinary cooperation, novel design, thorough preclinical clinical assessments. long-term unforeseen effects must also be considered from an ethical viewpoint. has considerable promise, but more research technological advances needed overcome limitations develop safe treatments.

Язык: Английский

Процитировано

0