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Bone-marrow-homing lipid nanoparticles for genome editing in diseased and malignant haematopoietic stem cells

The ability to genetically modify haematopoietic stem cells would allow the durable treatment of a diverse range of genetic disorders but gene delivery to the bone marrow has not been achieved. Here lipid nanoparticles that target and deliver mRNA to 14 unique cells within the bone marrow are presented.

Xizhen Lian
Sumanta Chatterjee
Daniel J. Siegwart
Research23 May 2024
Nature Nanotechnology

Volume: 19, P: 1409-1417
Selective organ targeting (SORT) nanoparticles for tissue-specific mRNA delivery and CRISPR–Cas gene editing

The addition of selective organ targeting molecules to nanoparticles allows the specific targeting of extrahepatic tissues, enabling gene editing of distinct cell populations outside the liver.

Qiang Cheng
Tuo Wei
Daniel J. Siegwart
Research06 Apr 2020
Nature Nanotechnology

Volume: 15, P: 313-320
Disrupting off-target Cas9 activity in the liver

Off-target genome editing in the liver can be reduced by using lipid nanoparticles to deliver oligonucleotides that disrupt the secondary structure of single-guide RNAs as well as short interfering RNAs targeting Cas9 mRNA.

Sean A. Dilliard
Daniel J. Siegwart
News & Views21 Feb 2022
Nature Biomedical Engineering

Volume: 6, P: 106-107
Passive, active and endogenous organ-targeted lipid and polymer nanoparticles for delivery of genetic drugs

Nucleic acids for gene silencing, expression and editing can precisely target disease at the molecular level but require effective delivery systems. This Review discusses the material and biological principles used to design delivery systems to target specific organs in the body.

Sean A. Dilliard
Daniel J. Siegwart
Reviews19 Jan 2023
Nature Reviews Materials

Volume: 8, P: 282-300

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Bone-marrow-homing lipid nanoparticles for genome editing in diseased and malignant haematopoietic stem cells

Selective organ targeting (SORT) nanoparticles for tissue-specific mRNA delivery and CRISPR–Cas gene editing

Disrupting off-target Cas9 activity in the liver

Passive, active and endogenous organ-targeted lipid and polymer nanoparticles for delivery of genetic drugs

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