A novel xenograft mouse model for testing approaches targeting human kappa light-chain diseases

Ma, Xiaobo; Zhou, Ping; Kugelmass, Adin; Toskic, Denis; Warner, Melissa; Lee, Lisa; Fogaren, Teresa; Godara, Amandeep; Wang, Ming; Li, Yamin; Yang, Liu; Xu, Qiaobing; Comenzo, Raymond L.

doi:10.1038/s41434-019-0070-y

Cited by 7 publications

(9 citation statements)

References 32 publications

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“…These LNPs were applied to an NOD scid gamma (NSG) JJN3 intraperitoneal mouse model. 8B-3 LNPs encapsulated with siRNA targeting the IGKC gene significantly reduced circulating Kappa light chains in the NSG JJN3 mice after three daily IP injections 32 , 33 .…”

Section: Lnps For the Delivery Of Nucleic Acidsmentioning

confidence: 94%

“…Our previous work primarily focused on the development of lipid materials and the examination in vitro in cultured cell lines and in vivo in small animal models 23 , 38 , 85 . So far, we have achieved successful long-term liver genome editing 36 , 37 , transient gene silencing 33 , 45 , 46 , tumor suppression 94 , 101 , and tissue regeneration 53 , 54 in mouse models. To facilitate the clinical translation of these promising systems, the top performing LNP systems identified from our previous studies will be further tested in large animal models and non-human primates.…”

Section: Challenges and Future Studiesmentioning

confidence: 99%

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Tailoring combinatorial lipid nanoparticles for intracellular delivery of nucleic acids, proteins, and drugs

Yang

et al. 2022

Acta Pharmaceutica Sinica B

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Section: Lnps For the Delivery Of Nucleic Acidsmentioning

confidence: 94%

Section: Challenges and Future Studiesmentioning

confidence: 99%

Tailoring combinatorial lipid nanoparticles for intracellular delivery of nucleic acids, proteins, and drugs

Yang

et al. 2022

Acta Pharmaceutica Sinica B

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“…Pharmacokinetic studies show that siRNA activity in vitro could last for almost a week, rendering weekly administration plausible [108]. Most recently, a group of investigators identified an anti-κLC CR siRNA that is safe and efficient in vitro with a reduction of circulating κ-LC for 8-12 days after administration, but more research is warranted [113].…”

Section: Treatment Strategies Targeting Immunoglobulin Mrnamentioning

confidence: 99%

Future Developments in the Treatment of AL Amyloidosis

Theodorakakou

Fotiou

Dimopoulos

2022

Hemato

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The treatment of AL amyloidosis has evolved, and outcomes have improved, but primarily for patients with low or intermediate-risk disease. Recent advances have been limited to improvements in anti-clonal therapies, which, alone, cannot change the poor prognosis of patients with high-risk disease. Thus, new strategies are needed that combine different approaches to the treatment of the disease. Targeted therapies against plasma/B-cell clones that avoid chemotherapy or potentially cardiotoxic drugs may improve the depth of hematologic responses and reduce complications. Amyloid fibril and light-chain oligomer targeting may reduce direct toxicity and enhance tissue clearance. Future combinations should be tailored to clone characteristics and specific amyloid properties, but early identification of those at high risk to develop AL amyloidosis will also be integrated into management algorithms.

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“…[88,186,195] Treatment opportunities also exist in the field of infections, [221][222][223] neurological disorders, [224][225][226] cardiovascular diseases, [227,228] and others. [9,13,18,229] Incorporating and delivering antibodies targeting these diseases/conditions can open up new possibilities for therapy.…”

Section: Delivery Of Cargo Antibodies With Therapeutic Potentialmentioning

confidence: 99%

Intracellular Antibody Delivery Mediated by Lipids, Polymers, and Inorganic Nanomaterials for Therapeutic Applications

et al. 2020

Advanced Therapeutics

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Therapeutic antibodies, due to their high affinity and specificity toward their biological targets, may demonstrate reduced harmful side effects compared with traditional drug moieties. While most of the as-yet clinically approved antibody therapeutics have targeted extracellular or membrane-bound domains, the ability to target intracellular antigens with antibodies opens up tremendous opportunities for imaging, diagnosis, and therapeutic applications. Generally, delivery concerns have limited the ability to target intracellular antigens, as many antibodies cannot easily cross the cell membrane due to their size and surface chemistry. Delivery platforms have been explored to address this issue, including physical methods, fusion protein/peptide techniques, and synthetic carrier-based systems. This review summarizes the progress of carrier-based intracellular antibody delivery systems employing synthetic lipids, polymers, and inorganic nanomaterials. Antibodies targeting various epitopes have been loaded through adsorption, conjugation, or physical encapsulation strategies. Successful intracellular deliveries have been demonstrated largely through fluorescence imaging using dye-labeled antibody cargos. Specific synthetic delivery platforms have great potential for ex vivo and in vivo therapeutic applications. Challenges and opportunities are further discussed for material scientists to explore in this research area.

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A novel xenograft mouse model for testing approaches targeting human kappa light-chain diseases

Cited by 7 publications

References 32 publications

Tailoring combinatorial lipid nanoparticles for intracellular delivery of nucleic acids, proteins, and drugs

Tailoring combinatorial lipid nanoparticles for intracellular delivery of nucleic acids, proteins, and drugs

Future Developments in the Treatment of AL Amyloidosis

Intracellular Antibody Delivery Mediated by Lipids, Polymers, and Inorganic Nanomaterials for Therapeutic Applications

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