Orchestrating a Symphony on a Single Conjugate: Aptamer Targeting, Gene Silencing, and Immunomodulation to Enhance Antitumor Response

Ng, Brandon D.; Peer, Dan

doi:10.1016/j.ymthe.2016.12.003

Cited by 4 publications

(3 citation statements)

References 16 publications

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“…The prostate specific membrane antigen (PSMA) RNA aptamer has become one of the most successful and widely used aptamer families to date 95 , 96 . Apart from safe and efficient delivery of RNAi payloads to specific target cells, leukocytes are notoriously hard to transfect 97 . Rajagopalan et al.…”

Section: Advantages and Approaches For Using Rna Nanotechnology To Di...mentioning

confidence: 99%

Targeted delivery of RNAi to cancer cells using RNA-ligand displaying exosome

Uddin

Binzel

Shu

et al. 2023

Acta Pharmaceutica Sinica B

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Section: Advantages and Approaches For Using Rna Nanotechnology To Di...mentioning

confidence: 99%

Targeted delivery of RNAi to cancer cells using RNA-ligand displaying exosome

Uddin

Binzel

Shu

et al. 2023

Acta Pharmaceutica Sinica B

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“…In an in vivo breast cancer model, the authors showed tumor infiltration by CD8 + T lymphocytes and an increase in tumor susceptibility to secondary treatment implemented, in parallel with the apt-siRNA conjugate. 32 Apt-short hairpin RNA (shRNA) conjugates were also engineered for lymphocyte targeting. Recently, Soldevilla et al 33 presented a CD40apt-shSMG1 for cancer immunotherapy.…”

Section: Apt-sirna Conjugatesmentioning

confidence: 99%

Current Progress in Non-viral RNAi-Based Delivery Strategies to Lymphocytes

et al. 2017

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RNAi-based therapy holds great promise, as it can be utilized for the treatment of multiple conditions in an accurate manner via sequence-specific manipulation of gene expression. To date, RNAi therapeutics have advanced into clinical trials for liver diseases and solid tumors; however, delivery of RNAi to leukocytes in general and to lymphocytes in particular remains a challenge. Lymphocytes are notoriously hard to transduce with RNAi payloads and are disseminated throughout the body, often located in deep tissues; therefore, developing an efficient systemic delivery system directed to lymphocytes is not a trivial task. Successful manipulation of lymphocyte function with RNAi possesses immense therapeutic potential, as it will enable researchers to resolve lymphocyte-implicated diseases such as inflammation, autoimmunity, transplant rejection, viral infections, and blood cancers. This potential has propelled the development of novel targeted delivery systems relying on the accumulating research knowledge from multiple disciplines, including materials science and engineering, immunology, and genetics. Here, we will discuss the recent progress in non-viral delivery strategies of RNAi payloads to lymphocytes. Special emphasis will be made on the challenges and potential opportunities in manipulating lymphocyte function with RNAi. These approaches might ultimately become a novel therapeutic modality to treat leukocyte-related diseases.

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“…So while in some cells the virus is easily eradicated, in others some indel mutations are refractory to recognition by the same gRNA as a result of changing the target DNA sequences, leading to the emergence of replication competent viruses that are resistant to Cas9/gRNA. More recently, Chaoran et al used an all-in-one adeno-associated virus (AAV) vector to deliver multiplex sgRNAs targeting four different viral structural genes and the Staphylococcus aureus-derived Cas9, demonstrating that this strategy greatly reduces the potential of HIV-1 escape and increases the possibility of HIV-1 excision despite the continuous mutations in the clinical HIV-1 patients’ population [ 48 ]. Many other therapies are currently ongoing to cure disease such as haemoglobinopathy, β-thalassaemia, Leber congenital amaurosis, haemophilia giving hope to all patients that could not count on alternative therapy ways [ 49 – 51 ].…”

Section: Clinical and Therapeutically Usesmentioning

confidence: 99%

Type II CRISPR/Cas9 approach in the oncological therapy

Biagioni

Chillà

Andreucci

et al. 2017

J Exp Clin Cancer Res

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CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) is a prokaryotic adaptable immune mechanism used by many bacteria and archaea to protect themselves from foreign nucleic acids. This complex system can recognize and cut non-self DNA in order to provide the prokaryotic organisms a strong defense against foreign viral or plasmid attacks and make the cell immune from further assaults. Today, it has been adapted to be used in vitro and in vivo in eukaryotic cells to perform a complete and highly selective gene knockout or a specific gene editing. The ease of use and the low cost are only two features that have made it very popular among the scientific community and the possibility to be used as a clinical treatment in several genetic derived pathologies has rapidly spread its fame worldwide. However, CRISPR is still not fully understood and many efforts need to be done in order to make it a real power tool for the human clinical treatment especially for oncological patients. Indeed, since cancer originates from non-lethal genetic disorders, CRISPR discovery fuels the hope to strike tumors on their roots. More than 4000 papers regarding CRISPR were published in the last ten years and only few of them take in count the possible applications in oncology. The purpose of this review is to clarify many problematics on the CRISPR usage and highlight its potential in oncological therapy.

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Orchestrating a Symphony on a Single Conjugate: Aptamer Targeting, Gene Silencing, and Immunomodulation to Enhance Antitumor Response

Cited by 4 publications

References 16 publications

Targeted delivery of RNAi to cancer cells using RNA-ligand displaying exosome

Targeted delivery of RNAi to cancer cells using RNA-ligand displaying exosome

Current Progress in Non-viral RNAi-Based Delivery Strategies to Lymphocytes

Type II CRISPR/Cas9 approach in the oncological therapy

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