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CRISPR Gene Editing
3 sources · 5 documents
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CRISPR Gene Editing Open →

Precise base editing of pathogenic variants in primary human T cells with engineered cytidine deaminases.

Authors: Lapinaite A, Doudna JA, Liu DR, Anzalone AV, Newby GA
Journal: Nature Biotechnology2026PubMed ↗

CRISPR base editors convert one DNA base into another without inducing double-strand breaks, but bystander edits at nearby positions limit therapeutic use. We engineer a cytidine deaminase variant with a narrowed editing window and demonstrate single-base precision at clinically relevant loci in primary human T cells.

Overview ↗

Genome-wide off-target activity profiling of CRISPR-Cas12a in primary human T cells.

Authors: Tsai SQ, Joung JK, Liu Q, Aryee MJ, Pinello L, Iafrate AJ
Journal: Nature Methods2026PubMed ↗

We extend CIRCLE-seq to map CRISPR-Cas12a off-target activity across the human genome at single-nucleotide resolution. Off-target rates fall an order of magnitude when guide RNAs are paired with high-fidelity Cas12a variants.

Overview ↗

Single-dose AAV delivery of adenine base editors restores dystrophin in a Duchenne muscular dystrophy model.

Authors: Newby GA, Yen JS, Woodard KJ, Mayuranathan T, Lazzarotto CR, Liu DR
Journal: Nature Medicine2026PubMed ↗

A single intramuscular injection of AAV9-packaged adenine base editors corrected a nonsense mutation in the dystrophin gene in a mouse model of Duchenne muscular dystrophy, restoring dystrophin protein expression to 60% of wild-type levels.

Overview ↗
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Open Tasks
CRISPR Gene Editing Open →
Read Liang et al., 2026 on AAV bystander editsFri
Re-run Tsai off-target analysis with v2 guidesOverdue
Email Newby lab re. AAV9 titer protocolToday
Draft Methods §2.3, delivery comparisonMon
Cite Lapinaite 2026 in Methods §1Tue
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mRNA Vaccine Design Open →
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Genome Lab▾ / CRISPR Gene Editing▾
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find a paper that bridges my AAV delivery and off-target specificity work

Your three papers cover both ends — off-target specificity and AAV delivery — but none connect them. Liang 2026 does: off-target profiling after in-vivo AAV delivery, and it isn't in your library yet. Here's how they line up:

PaperFocusSystemKey result
Lapinaite 2026Narrowed-window base editorHuman T cellsSingle-base precision, fewer bystander edits
Tsai 2026Genome-wide off-target mapCIRCLE-seq, in vitroOff-targets ↓ 10× with hi-fi Cas12a
Newby 2026AAV base-editor deliveryDMD mouse, in vivo60% dystrophin from a single dose
Liang 2026Off-target after AAV deliveryMouse liver, in vivoBystander edits at 4× the RNP rate
save it to my library

Done — added Liang 2026 and tagged it AAV delivery and off-target.

Library updates
Added: Off-target edits accumulate after in-vivo AAV delivery
Tagged AAV delivery, off-target · CRISPR Gene Editing
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PubMed 2016

Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage.

Authors: Komor AC, Kim YB, Packer MS, Zuris JA, Liu DR
Journal: NatureDOI 10.1038/nature17946

Current genome-editing technologies introduce double-stranded DNA breaks at a target locus as the first step to gene correction. Although most genetic diseases arise from point mutations, current approaches to point-mutation correction are inefficient and typically induce an abundance of random insertions and deletions.

Overview ↗
PubMed 2017

Programmable base editing of A•T to G•C in genomic DNA without DNA cleavage.

Authors: Gaudelli NM, Komor AC, Rees HA, Packer MS, Badran AH, Bryson DI
Journal: NatureDOI 10.1038/nature24644

The spontaneous deamination of cytosine is a major source of transitions from C•G to T•A base pairs, which account for half of known pathogenic point mutations in humans. The ability to efficiently convert targeted A•T base pairs to G•C could therefore advance the study and treatment of genetic diseases.

Overview ↗
PubMed 2015

GUIDE-seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases.

Authors: Tsai SQ, Zheng Z, Nguyen NT, Liebers M, Topkar VV, Thapar V
Journal: Nature BiotechnologyDOI 10.1038/nbt.3117

CRISPR RNA-guided nucleases are widely used genome-editing reagents, but methods to delineate their genome-wide, off-target cleavage activities have been lacking. Here we describe GUIDE-seq, an approach for global detection of DNA double-stranded breaks introduced by RGNs and potentially other nucleases.

Overview ↗
Documents
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Tasks
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Task Priority Status Due
Read Liang et al., 2026 on AAV bystander edits
High
In progress
Fri, May 29
Re-run Tsai off-target analysis with v2 guides
High
In progress
Wed, May 21
Draft Methods §2.3, delivery comparison
Medium
To do
Mon, Jun 2
Email Newby lab re. AAV9 titer protocol
Medium
To do
Today
Cite Lapinaite 2026 in Methods §1
Low
To do
Tue, May 26
Pull Anzalone 2024 prime-editor variants from Zotero
Low
Done
Mon, May 19
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Priority
Status
mm/dd/yyyy
To do 3
Draft Methods §2.3, delivery comparison
Medium-Mon, Jun 2
Email Newby lab re. AAV9 titer protocol
Medium-Today
Cite Lapinaite 2026 in Methods §1
Low-Tue, May 26
+ Add task
In progress 2
Read Liang et al., 2026 on AAV bystander edits
High-Fri, May 29
Re-run Tsai off-target analysis with v2 guides
High-Wed, May 21
+ Add task
Done 1
Pull Anzalone 2024 prime-editor variants from Zotero
Low-Mon, May 19
+ Add task
May 19May 26Jun 2Jun 9
Read Liang et al., 2026 on AAV bystander edits
Re-run Tsai off-target analysis with v2 guides
Draft Methods §2.3, delivery comparison
Email Newby lab re. AAV9 titer protocol
Cite Lapinaite 2026 in Methods §1
Pull Anzalone 2024 prime-editor variants from Zotero
Daily Briefing

Precise base editing of pathogenic variants in primary human T cells with engineered cytidine deaminases.

Authors: Lapinaite A, Doudna JA, Liu DR, Anzalone AV, Newby GA
Journal: Nature Biotechnology2026PubMed ↗

CRISPR base editors convert one DNA base into another without inducing double-strand breaks, but bystander edits at nearby positions limit therapeutic use. We engineer a cytidine deaminase variant with a narrowed editing window and demonstrate single-base precision at clinically relevant loci in primary human T cells.

Overview ↗

Genome-wide off-target activity profiling of CRISPR-Cas12a in primary human T cells.

Authors: Tsai SQ, Joung JK, Liu Q, Aryee MJ, Pinello L, Iafrate AJ
Journal: Nature Methods2026PubMed ↗

We extend CIRCLE-seq to map CRISPR-Cas12a off-target activity across the human genome at single-nucleotide resolution. Off-target rates fall an order of magnitude when guide RNAs are paired with high-fidelity Cas12a variants.

Overview ↗

Single-dose AAV delivery of adenine base editors restores dystrophin in a Duchenne muscular dystrophy model.

Authors: Newby GA, Yen JS, Woodard KJ, Mayuranathan T, Lazzarotto CR, Liu DR
Journal: Nature Medicine2026PubMed ↗

A single intramuscular injection of AAV9-packaged adenine base editors corrected a nonsense mutation in the dystrophin gene in a mouse model of Duchenne muscular dystrophy, restoring dystrophin protein expression to 60% of wild-type levels.

Overview ↗

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To do 3
Email advisor about the methods section
MediumJun 12
Re-run the power analysis with n = 240
HighJun 13
Pull DOIs for the introduction citations
LowJun 16
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In progress 2
Draft Results §3.2, genotype frequencies
HighJun 11
Reconcile Table 2 with the raw export
MediumJun 15
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Done 2
Import Lapinaite 2026 into the library
LowJun 8
Outline the discussion section
MediumJun 6
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