ArticleACS nano2023
Ionizable Lipid Nanoparticles for Therapeutic Base Editing of Congenital Brain Disease.
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Article in ACS nano, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 47 papers.
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47citing papers in PubMed
9.7field-weighted citation impact, top 1% of its field
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Who cites it
47 citing papers in PubMed, 63 citations in OpenAlex.
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- Genome-wide CRISPR screening identifies cellular factors controlling nonviral genome editing efficiency.Nature communications · 2026Article
- Nanoparticle-assisted gene editing for genomic disorders in the central nervous system.Neural regeneration research · 2026Article
- sgRNA amount is a limiting factor in adenine base editing using RNA LNPs.Molecular therapy. Nucleic acids · 2026Article
- Optimizing C14120-based lipid nanoparticles for mRNA delivery: Editorial commentary.Molecular therapy. Nucleic acids · 2026Article
- PEG-associated efficacy loss in lipid nanoparticles persists across formulation modifications for mRNA delivery.Research square · 2026Article
- Cholesterol analogs modulate lipid nanoparticle performance for mRNA delivery after lyophilization and enable ocular disease therapy.Materials today. Bio · 2026Article
- The RNA delivery dilemma-lipid versus polymer nanoparticle platforms.Drug delivery and translational research · 2026Article
- Programmable lipid nanoparticles for RNA therapeutics: Design principles and clinical translation.Materials today. Bio · 2026Review
- Bioconjugates for improved delivery of oligonucleotide therapeutics to the central nervous system.Advanced drug delivery reviews · 2026Review
- Oligodendrocyte: Development, Plasticity, Biological Functions, Diseases, and Therapeutic Targets.MedComm · 2026Review
- Advancements in Neonatal Brain Injury Treatment: Nanomedicine-Based Strategies.International journal of nanomedicine · 2026Review
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- Lipid Nanoparticles for Delivery of CRISPR Gene Editing Components.Small methods · 2026Review
- Systemic mRNA-LNP administration in fetuses improves survival in a mouse model of spinal muscular atrophy.Molecular therapy. Methods & clinical development · 2025Article
- Modified self-amplifying RNA mediates robust and prolonged gene expression in the mammalian brain.bioRxiv : the preprint server for biology · 2025Article
- Sonochemical synthesis of nanoparticles from bioactive compounds: advances, challenges, and future perspectives.Ultrasonics sonochemistry · 2025Review
- Advancing Gene Therapy for Phenylketonuria: From Precision Editing to Clinical Translation.International journal of molecular sciences · 2025Review
Corrections and comments
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Authors and funding
28 authors at 5 institutions in 2 countries.
Rohan PalankiDepartment of Bioengineering, University of Pennsylvania, Philadelphia, Pennsylvania 19104, United States.ORCID 0000-0001-5168-5634
Sourav K BoseCenter for Fetal Research, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania 19104, United States.
Apeksha DaveCenter for Fetal Research, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania 19104, United States.
Brandon M WhiteCenter for Fetal Research, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania 19104, United States.
Cara BerkowitzCenter for Fetal Research, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania 19104, United States.
Valerie LuksCenter for Fetal Research, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania 19104, United States.
Fazeela YaqoobDepartment of Psychiatry, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania 19104, United States.
Emily HanDepartment of Bioengineering, University of Pennsylvania, Philadelphia, Pennsylvania 19104, United States.
Kelsey L SwingleDepartment of Bioengineering, University of Pennsylvania, Philadelphia, Pennsylvania 19104, United States.ORCID 0000-0001-8475-9206
Pallavi MenonCenter for Fetal Research, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania 19104, United States.
Emily HodgsonCenter for Fetal Research, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania 19104, United States.
Arijit BiswasDuke-NUS Graduate Medical School, Singapore, 169547, Singapore.
Margaret M BillingsleyDepartment of Bioengineering, University of Pennsylvania, Philadelphia, Pennsylvania 19104, United States.ORCID 0000-0003-4499-9066
Li LiCardiovascular Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania 19104, United States.
Fan YipingDuke-NUS Graduate Medical School, Singapore, 169547, Singapore.
Marco CarpenterCenter for Fetal Research, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania 19104, United States.
Alexandra TrokhanCenter for Fetal Research, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania 19104, United States.
Julie YeoDuke-NUS Graduate Medical School, Singapore, 169547, Singapore.
Nuryanti JohanaDuke-NUS Graduate Medical School, Singapore, 169547, Singapore.
Tan Yi WanDuke-NUS Graduate Medical School, Singapore, 169547, Singapore.
Mohamad-Gabriel AlamehDepartment of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania 19104, United States.ORCID 0000-0002-5672-6930
Frederick Chris BennettDepartment of Psychiatry, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania 19104, United States.
Phillip B StormDivision of Neurosurgery, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania 19104, United States.
Rajan JainDepartment of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania 19104, United States.
Jerry ChanDuke-NUS Graduate Medical School, Singapore, 169547, Singapore.
Drew WeissmanDepartment of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania 19104, United States.ORCID 0000-0002-1501-6510
Michael J MitchellDepartment of Bioengineering, University of Pennsylvania, Philadelphia, Pennsylvania 19104, United States.ORCID 0000-0002-3628-2244
William H PeranteauCenter for Fetal Research, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania 19104, United States.
Children's Hospital of Philadelphia · USUniversity of Pennsylvania · USDuke-NUS Medical School · SGCalifornia Institute for Regenerative Medicine · USKK Women's and Children's Hospital · SG
Funding
MEDICAL SCIENTIST TRAINING PROGRAMT32GM007170 · NIGMS · UNIVERSITY OF PENNSYLVANIA · PI BRASS, LAWRENCE F · 1985 to 2022
$54.0MIn utero gene editing to cure a metabolic liver diseaseR01DK123049 · NIDDK · CHILDREN'S HOSP OF PHILADELPHIA · PI PERANTEAU, WILLIAM H. · 2020 to 2024
$3.7MDeciphering how 3D genome organization orchestrates cardiac cellular identityR35HL166663 · NHLBI · UNIVERSITY OF PENNSYLVANIA · PI Rajan Jain · 2023 to 2026
$3.5MIn utero CRISPR-mediated gene editing to cure congenital monogenic disordersDP2HL152427 · NHLBI · CHILDREN'S HOSP OF PHILADELPHIA · PI PERANTEAU, WILLIAM H. · 2019 to 2019
$2.6MA data-driven drug delivery (4D) platform for probing and treating the chemoresistant bone marrow microenvironmentDP2TR002776 · NCATS · UNIVERSITY OF PENNSYLVANIA · PI MITCHELL, MICHAEL J · 2018 to 2018
$2.4MIonizable Lipid Nanoparticles for Fetal Lung TargetingF30HL162465 · NHLBI · UNIVERSITY OF PENNSYLVANIA · PI PALANKI, ROHAN · 2022 to 2025
$178kNCATS NIH HHS DP2 TR002776NHLBI NIH HHS DP2 HL152427NHLBI NIH HHS F30 HL162465NIDDK NIH HHS R01 DK123049NIGMS NIH HHS T32 GM007170
Abstract
Delivery of mRNA-based therapeutics to the perinatal brain holds great potential in treating congenital brain diseases. However, nonviral delivery platforms that facilitate nucleic acid delivery in this environment have yet to be rigorously studied. Here, we screen a diverse library of ionizable lipid nanoparticles (LNPs) via intracerebroventricular (ICV) injection in both fetal and neonatal mice and identify an LNP formulation with greater functional mRNA delivery in the perinatal brain than an FDA-approved industry standard LNP. Following
Indexed as
Brain DiseasesNanoparticlesAnimalsGene EditingHumansLipidsLiposomesMiceRNA, MessengerRNA, Small InterferingLipid NanoparticlesLipidsLiposomesRNA, MessengerRNA, Small Interferingcongenital brain diseasefetal gene therapygene editingionizable lipid nanoparticlesmRNA delivery
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.