Source 1primary paper2021Communications Biology
Toolkit/SpCas9-NG
SpCas9-NG
Also known as: engineered SpCas9 nuclease variant, SpCas9-NG-mediated gene editing
Taxonomy: Technique Branch / Method. Workflows sit above the mechanism and technique branches rather than replacing them.
Summary
SpCas9-NG is an engineered Streptococcus pyogenes Cas9 nuclease variant that recognizes NGN protospacer-adjacent motifs instead of the NGG PAM required by wild-type SpCas9. In the cited 2021 Communications Biology study, it was used for RNA-guided genome editing to target the boundary of expanded CAG repeats and induce precise repeat contraction in a Huntington’s disease mouse model context.
Usefulness & Problems
Why this is useful
SpCas9-NG is useful because its broadened NGN PAM recognition expands access to genomic sites that are difficult to target with wild-type SpCas9. The cited study specifically positions it as a tool for repairing abnormally expanded CAG repeats and other disease mutations that are otherwise poorly accessible to WT-SpCas9.
Source:
Our study shows that SpCas9-NG can be a powerful tool for repairing abnormally expanded CAG repeats as well as other disease mutations that are difficult to access with WT-SpCas9.
Problem solved
This tool addresses the PAM restriction of wild-type SpCas9, which limits editing at disease-relevant loci lacking a nearby NGG PAM. In the supplied study, that expanded targeting scope enabled editing at the boundary of pathogenic CAG repeat tracts in Huntington’s disease model cells.
Source:
Our study shows that SpCas9-NG can be a powerful tool for repairing abnormally expanded CAG repeats as well as other disease mutations that are difficult to access with WT-SpCas9.
Taxonomy & Function
Primary hierarchy
Technique Branch
Method: A concrete method used to build, optimize, or evolve an engineered system.
Mechanisms
genome editing via nuclease-mediated dna cleavagegenome editing via nuclease-mediated dna cleavagepam recognition broadeningpam recognition broadeningrna-guided dna targetingrna-guided dna targetingTechniques
No technique tags yet.
Target processes
No target processes tagged yet.
Implementation Constraints
cofactor dependency: cofactor requirement unknownencoding mode: genetically encodedimplementation constraint: context specific validationoperating role: builder
The cited application used SpCas9-NG for targeting the boundary of CAG repeats in HD-mouse-derived embryonic stem cells, followed by assessment in differentiated neurons and animals derived from repaired ES cells. Beyond its identity as an engineered SpCas9 nuclease variant with NGN PAM recognition, the supplied evidence does not specify construct architecture, guide design rules, delivery modality, or expression system details.
The provided evidence is centered on a single disease-model application in Huntington’s disease and does not report broader benchmarking across loci, cell types, or organisms. The supplied material also does not provide quantitative editing efficiency, off-target profiles, delivery constraints, or direct comparisons with alternative PAM-relaxed Cas9 variants.
Validation
Cell-freeBacteriaMammalianMouseHumanTherapeuticIndep. Replication
Observations
successMouseapplication demoHD-mouse-derived embryonic stem cells
Inferred from claim c3 during normalization. Targeting the boundary of CAG repeats with SpCas9-NG precisely contracted abnormally expanded CAG repeat tracts in HD-mouse-derived embryonic stem cells. Derived from claim c3. Quoted text: By targeting the boundary of CAG repeats with SpCas9-NG, we precisely contracted the repeat tracts in HD-mouse-derived embryonic stem (ES) cells.
Source:
successMouseapplication demoHD-mouse-derived embryonic stem cells
Inferred from claim c3 during normalization. Targeting the boundary of CAG repeats with SpCas9-NG precisely contracted abnormally expanded CAG repeat tracts in HD-mouse-derived embryonic stem cells. Derived from claim c3. Quoted text: By targeting the boundary of CAG repeats with SpCas9-NG, we precisely contracted the repeat tracts in HD-mouse-derived embryonic stem (ES) cells.
Source:
successMouseapplication demoHD-mouse-derived embryonic stem cells
Inferred from claim c3 during normalization. Targeting the boundary of CAG repeats with SpCas9-NG precisely contracted abnormally expanded CAG repeat tracts in HD-mouse-derived embryonic stem cells. Derived from claim c3. Quoted text: By targeting the boundary of CAG repeats with SpCas9-NG, we precisely contracted the repeat tracts in HD-mouse-derived embryonic stem (ES) cells.
Source:
successMouseapplication demoHD-mouse-derived embryonic stem cells
Inferred from claim c3 during normalization. Targeting the boundary of CAG repeats with SpCas9-NG precisely contracted abnormally expanded CAG repeat tracts in HD-mouse-derived embryonic stem cells. Derived from claim c3. Quoted text: By targeting the boundary of CAG repeats with SpCas9-NG, we precisely contracted the repeat tracts in HD-mouse-derived embryonic stem (ES) cells.
Source:
successMouseapplication demoHD-mouse-derived embryonic stem cells
Inferred from claim c3 during normalization. Targeting the boundary of CAG repeats with SpCas9-NG precisely contracted abnormally expanded CAG repeat tracts in HD-mouse-derived embryonic stem cells. Derived from claim c3. Quoted text: By targeting the boundary of CAG repeats with SpCas9-NG, we precisely contracted the repeat tracts in HD-mouse-derived embryonic stem (ES) cells.
Source:
successMouseapplication demoHD-mouse-derived embryonic stem cells
Inferred from claim c3 during normalization. Targeting the boundary of CAG repeats with SpCas9-NG precisely contracted abnormally expanded CAG repeat tracts in HD-mouse-derived embryonic stem cells. Derived from claim c3. Quoted text: By targeting the boundary of CAG repeats with SpCas9-NG, we precisely contracted the repeat tracts in HD-mouse-derived embryonic stem (ES) cells.
Source:
successMouseapplication demoHD-mouse-derived embryonic stem cells
Inferred from claim c3 during normalization. Targeting the boundary of CAG repeats with SpCas9-NG precisely contracted abnormally expanded CAG repeat tracts in HD-mouse-derived embryonic stem cells. Derived from claim c3. Quoted text: By targeting the boundary of CAG repeats with SpCas9-NG, we precisely contracted the repeat tracts in HD-mouse-derived embryonic stem (ES) cells.
Source:
Supporting Sources
Ranked Claims
SpCas9-NG can be a powerful tool for repairing abnormally expanded CAG repeats and other disease mutations that are difficult to access with WT-SpCas9.
Our study shows that SpCas9-NG can be a powerful tool for repairing abnormally expanded CAG repeats as well as other disease mutations that are difficult to access with WT-SpCas9.
SpCas9-NG can be a powerful tool for repairing abnormally expanded CAG repeats and other disease mutations that are difficult to access with WT-SpCas9.
Our study shows that SpCas9-NG can be a powerful tool for repairing abnormally expanded CAG repeats as well as other disease mutations that are difficult to access with WT-SpCas9.
SpCas9-NG can be a powerful tool for repairing abnormally expanded CAG repeats and other disease mutations that are difficult to access with WT-SpCas9.
Our study shows that SpCas9-NG can be a powerful tool for repairing abnormally expanded CAG repeats as well as other disease mutations that are difficult to access with WT-SpCas9.
SpCas9-NG can be a powerful tool for repairing abnormally expanded CAG repeats and other disease mutations that are difficult to access with WT-SpCas9.
Our study shows that SpCas9-NG can be a powerful tool for repairing abnormally expanded CAG repeats as well as other disease mutations that are difficult to access with WT-SpCas9.
SpCas9-NG can be a powerful tool for repairing abnormally expanded CAG repeats and other disease mutations that are difficult to access with WT-SpCas9.
Our study shows that SpCas9-NG can be a powerful tool for repairing abnormally expanded CAG repeats as well as other disease mutations that are difficult to access with WT-SpCas9.
SpCas9-NG can be a powerful tool for repairing abnormally expanded CAG repeats and other disease mutations that are difficult to access with WT-SpCas9.
Our study shows that SpCas9-NG can be a powerful tool for repairing abnormally expanded CAG repeats as well as other disease mutations that are difficult to access with WT-SpCas9.
SpCas9-NG can be a powerful tool for repairing abnormally expanded CAG repeats and other disease mutations that are difficult to access with WT-SpCas9.
Our study shows that SpCas9-NG can be a powerful tool for repairing abnormally expanded CAG repeats as well as other disease mutations that are difficult to access with WT-SpCas9.
SpCas9-NG can be a powerful tool for repairing abnormally expanded CAG repeats and other disease mutations that are difficult to access with WT-SpCas9.
Our study shows that SpCas9-NG can be a powerful tool for repairing abnormally expanded CAG repeats as well as other disease mutations that are difficult to access with WT-SpCas9.
SpCas9-NG can be a powerful tool for repairing abnormally expanded CAG repeats and other disease mutations that are difficult to access with WT-SpCas9.
Our study shows that SpCas9-NG can be a powerful tool for repairing abnormally expanded CAG repeats as well as other disease mutations that are difficult to access with WT-SpCas9.
SpCas9-NG can be a powerful tool for repairing abnormally expanded CAG repeats and other disease mutations that are difficult to access with WT-SpCas9.
Our study shows that SpCas9-NG can be a powerful tool for repairing abnormally expanded CAG repeats as well as other disease mutations that are difficult to access with WT-SpCas9.
SpCas9-NG can be a powerful tool for repairing abnormally expanded CAG repeats and other disease mutations that are difficult to access with WT-SpCas9.
Our study shows that SpCas9-NG can be a powerful tool for repairing abnormally expanded CAG repeats as well as other disease mutations that are difficult to access with WT-SpCas9.
SpCas9-NG can be a powerful tool for repairing abnormally expanded CAG repeats and other disease mutations that are difficult to access with WT-SpCas9.
Our study shows that SpCas9-NG can be a powerful tool for repairing abnormally expanded CAG repeats as well as other disease mutations that are difficult to access with WT-SpCas9.
SpCas9-NG can be a powerful tool for repairing abnormally expanded CAG repeats and other disease mutations that are difficult to access with WT-SpCas9.
Our study shows that SpCas9-NG can be a powerful tool for repairing abnormally expanded CAG repeats as well as other disease mutations that are difficult to access with WT-SpCas9.
SpCas9-NG can be a powerful tool for repairing abnormally expanded CAG repeats and other disease mutations that are difficult to access with WT-SpCas9.
Our study shows that SpCas9-NG can be a powerful tool for repairing abnormally expanded CAG repeats as well as other disease mutations that are difficult to access with WT-SpCas9.
SpCas9-NG can be a powerful tool for repairing abnormally expanded CAG repeats and other disease mutations that are difficult to access with WT-SpCas9.
Our study shows that SpCas9-NG can be a powerful tool for repairing abnormally expanded CAG repeats as well as other disease mutations that are difficult to access with WT-SpCas9.
SpCas9-NG can be a powerful tool for repairing abnormally expanded CAG repeats and other disease mutations that are difficult to access with WT-SpCas9.
Our study shows that SpCas9-NG can be a powerful tool for repairing abnormally expanded CAG repeats as well as other disease mutations that are difficult to access with WT-SpCas9.
SpCas9-NG can be a powerful tool for repairing abnormally expanded CAG repeats and other disease mutations that are difficult to access with WT-SpCas9.
Our study shows that SpCas9-NG can be a powerful tool for repairing abnormally expanded CAG repeats as well as other disease mutations that are difficult to access with WT-SpCas9.
Targeting the boundary of CAG repeats with SpCas9-NG precisely contracted abnormally expanded CAG repeat tracts in HD-mouse-derived embryonic stem cells.
By targeting the boundary of CAG repeats with SpCas9-NG, we precisely contracted the repeat tracts in HD-mouse-derived embryonic stem (ES) cells.
Targeting the boundary of CAG repeats with SpCas9-NG precisely contracted abnormally expanded CAG repeat tracts in HD-mouse-derived embryonic stem cells.
By targeting the boundary of CAG repeats with SpCas9-NG, we precisely contracted the repeat tracts in HD-mouse-derived embryonic stem (ES) cells.
Targeting the boundary of CAG repeats with SpCas9-NG precisely contracted abnormally expanded CAG repeat tracts in HD-mouse-derived embryonic stem cells.
By targeting the boundary of CAG repeats with SpCas9-NG, we precisely contracted the repeat tracts in HD-mouse-derived embryonic stem (ES) cells.
Targeting the boundary of CAG repeats with SpCas9-NG precisely contracted abnormally expanded CAG repeat tracts in HD-mouse-derived embryonic stem cells.
By targeting the boundary of CAG repeats with SpCas9-NG, we precisely contracted the repeat tracts in HD-mouse-derived embryonic stem (ES) cells.
Targeting the boundary of CAG repeats with SpCas9-NG precisely contracted abnormally expanded CAG repeat tracts in HD-mouse-derived embryonic stem cells.
By targeting the boundary of CAG repeats with SpCas9-NG, we precisely contracted the repeat tracts in HD-mouse-derived embryonic stem (ES) cells.
Targeting the boundary of CAG repeats with SpCas9-NG precisely contracted abnormally expanded CAG repeat tracts in HD-mouse-derived embryonic stem cells.
By targeting the boundary of CAG repeats with SpCas9-NG, we precisely contracted the repeat tracts in HD-mouse-derived embryonic stem (ES) cells.
Targeting the boundary of CAG repeats with SpCas9-NG precisely contracted abnormally expanded CAG repeat tracts in HD-mouse-derived embryonic stem cells.
By targeting the boundary of CAG repeats with SpCas9-NG, we precisely contracted the repeat tracts in HD-mouse-derived embryonic stem (ES) cells.
Targeting the boundary of CAG repeats with SpCas9-NG precisely contracted abnormally expanded CAG repeat tracts in HD-mouse-derived embryonic stem cells.
By targeting the boundary of CAG repeats with SpCas9-NG, we precisely contracted the repeat tracts in HD-mouse-derived embryonic stem (ES) cells.
Targeting the boundary of CAG repeats with SpCas9-NG precisely contracted abnormally expanded CAG repeat tracts in HD-mouse-derived embryonic stem cells.
By targeting the boundary of CAG repeats with SpCas9-NG, we precisely contracted the repeat tracts in HD-mouse-derived embryonic stem (ES) cells.
Targeting the boundary of CAG repeats with SpCas9-NG precisely contracted abnormally expanded CAG repeat tracts in HD-mouse-derived embryonic stem cells.
By targeting the boundary of CAG repeats with SpCas9-NG, we precisely contracted the repeat tracts in HD-mouse-derived embryonic stem (ES) cells.
Targeting the boundary of CAG repeats with SpCas9-NG precisely contracted abnormally expanded CAG repeat tracts in HD-mouse-derived embryonic stem cells.
By targeting the boundary of CAG repeats with SpCas9-NG, we precisely contracted the repeat tracts in HD-mouse-derived embryonic stem (ES) cells.
Targeting the boundary of CAG repeats with SpCas9-NG precisely contracted abnormally expanded CAG repeat tracts in HD-mouse-derived embryonic stem cells.
By targeting the boundary of CAG repeats with SpCas9-NG, we precisely contracted the repeat tracts in HD-mouse-derived embryonic stem (ES) cells.
Targeting the boundary of CAG repeats with SpCas9-NG precisely contracted abnormally expanded CAG repeat tracts in HD-mouse-derived embryonic stem cells.
By targeting the boundary of CAG repeats with SpCas9-NG, we precisely contracted the repeat tracts in HD-mouse-derived embryonic stem (ES) cells.
Targeting the boundary of CAG repeats with SpCas9-NG precisely contracted abnormally expanded CAG repeat tracts in HD-mouse-derived embryonic stem cells.
By targeting the boundary of CAG repeats with SpCas9-NG, we precisely contracted the repeat tracts in HD-mouse-derived embryonic stem (ES) cells.
Targeting the boundary of CAG repeats with SpCas9-NG precisely contracted abnormally expanded CAG repeat tracts in HD-mouse-derived embryonic stem cells.
By targeting the boundary of CAG repeats with SpCas9-NG, we precisely contracted the repeat tracts in HD-mouse-derived embryonic stem (ES) cells.
Targeting the boundary of CAG repeats with SpCas9-NG precisely contracted abnormally expanded CAG repeat tracts in HD-mouse-derived embryonic stem cells.
By targeting the boundary of CAG repeats with SpCas9-NG, we precisely contracted the repeat tracts in HD-mouse-derived embryonic stem (ES) cells.
Targeting the boundary of CAG repeats with SpCas9-NG precisely contracted abnormally expanded CAG repeat tracts in HD-mouse-derived embryonic stem cells.
By targeting the boundary of CAG repeats with SpCas9-NG, we precisely contracted the repeat tracts in HD-mouse-derived embryonic stem (ES) cells.
Phenotypic abnormalities were recovered in differentiated neurons and in animals produced from repaired ES cells.
we confirmed the recovery of phenotypic abnormalities in differentiated neurons and animals produced from repaired ES cells
Phenotypic abnormalities were recovered in differentiated neurons and in animals produced from repaired ES cells.
we confirmed the recovery of phenotypic abnormalities in differentiated neurons and animals produced from repaired ES cells
Phenotypic abnormalities were recovered in differentiated neurons and in animals produced from repaired ES cells.
we confirmed the recovery of phenotypic abnormalities in differentiated neurons and animals produced from repaired ES cells
Phenotypic abnormalities were recovered in differentiated neurons and in animals produced from repaired ES cells.
we confirmed the recovery of phenotypic abnormalities in differentiated neurons and animals produced from repaired ES cells
Phenotypic abnormalities were recovered in differentiated neurons and in animals produced from repaired ES cells.
we confirmed the recovery of phenotypic abnormalities in differentiated neurons and animals produced from repaired ES cells
Phenotypic abnormalities were recovered in differentiated neurons and in animals produced from repaired ES cells.
we confirmed the recovery of phenotypic abnormalities in differentiated neurons and animals produced from repaired ES cells
Phenotypic abnormalities were recovered in differentiated neurons and in animals produced from repaired ES cells.
we confirmed the recovery of phenotypic abnormalities in differentiated neurons and animals produced from repaired ES cells
Phenotypic abnormalities were recovered in differentiated neurons and in animals produced from repaired ES cells.
we confirmed the recovery of phenotypic abnormalities in differentiated neurons and animals produced from repaired ES cells
Phenotypic abnormalities were recovered in differentiated neurons and in animals produced from repaired ES cells.
we confirmed the recovery of phenotypic abnormalities in differentiated neurons and animals produced from repaired ES cells
Phenotypic abnormalities were recovered in differentiated neurons and in animals produced from repaired ES cells.
we confirmed the recovery of phenotypic abnormalities in differentiated neurons and animals produced from repaired ES cells
Phenotypic abnormalities were recovered in differentiated neurons and in animals produced from repaired ES cells.
we confirmed the recovery of phenotypic abnormalities in differentiated neurons and animals produced from repaired ES cells
Phenotypic abnormalities were recovered in differentiated neurons and in animals produced from repaired ES cells.
we confirmed the recovery of phenotypic abnormalities in differentiated neurons and animals produced from repaired ES cells
Phenotypic abnormalities were recovered in differentiated neurons and in animals produced from repaired ES cells.
we confirmed the recovery of phenotypic abnormalities in differentiated neurons and animals produced from repaired ES cells
Phenotypic abnormalities were recovered in differentiated neurons and in animals produced from repaired ES cells.
we confirmed the recovery of phenotypic abnormalities in differentiated neurons and animals produced from repaired ES cells
Phenotypic abnormalities were recovered in differentiated neurons and in animals produced from repaired ES cells.
we confirmed the recovery of phenotypic abnormalities in differentiated neurons and animals produced from repaired ES cells
Phenotypic abnormalities were recovered in differentiated neurons and in animals produced from repaired ES cells.
we confirmed the recovery of phenotypic abnormalities in differentiated neurons and animals produced from repaired ES cells
Phenotypic abnormalities were recovered in differentiated neurons and in animals produced from repaired ES cells.
we confirmed the recovery of phenotypic abnormalities in differentiated neurons and animals produced from repaired ES cells
WT-SpCas9 requires an NGG PAM for target recognition, which restricts targetable disease mutations.
the widely used Streptococcus pyogenes Cas9 (WT-SpCas9) requires an NGG protospacer adjacent motif (PAM) for target recognition, thereby restricting targetable disease mutations
WT-SpCas9 requires an NGG PAM for target recognition, which restricts targetable disease mutations.
the widely used Streptococcus pyogenes Cas9 (WT-SpCas9) requires an NGG protospacer adjacent motif (PAM) for target recognition, thereby restricting targetable disease mutations
WT-SpCas9 requires an NGG PAM for target recognition, which restricts targetable disease mutations.
the widely used Streptococcus pyogenes Cas9 (WT-SpCas9) requires an NGG protospacer adjacent motif (PAM) for target recognition, thereby restricting targetable disease mutations
WT-SpCas9 requires an NGG PAM for target recognition, which restricts targetable disease mutations.
the widely used Streptococcus pyogenes Cas9 (WT-SpCas9) requires an NGG protospacer adjacent motif (PAM) for target recognition, thereby restricting targetable disease mutations
WT-SpCas9 requires an NGG PAM for target recognition, which restricts targetable disease mutations.
the widely used Streptococcus pyogenes Cas9 (WT-SpCas9) requires an NGG protospacer adjacent motif (PAM) for target recognition, thereby restricting targetable disease mutations
WT-SpCas9 requires an NGG PAM for target recognition, which restricts targetable disease mutations.
the widely used Streptococcus pyogenes Cas9 (WT-SpCas9) requires an NGG protospacer adjacent motif (PAM) for target recognition, thereby restricting targetable disease mutations
WT-SpCas9 requires an NGG PAM for target recognition, which restricts targetable disease mutations.
the widely used Streptococcus pyogenes Cas9 (WT-SpCas9) requires an NGG protospacer adjacent motif (PAM) for target recognition, thereby restricting targetable disease mutations
WT-SpCas9 requires an NGG PAM for target recognition, which restricts targetable disease mutations.
the widely used Streptococcus pyogenes Cas9 (WT-SpCas9) requires an NGG protospacer adjacent motif (PAM) for target recognition, thereby restricting targetable disease mutations
WT-SpCas9 requires an NGG PAM for target recognition, which restricts targetable disease mutations.
the widely used Streptococcus pyogenes Cas9 (WT-SpCas9) requires an NGG protospacer adjacent motif (PAM) for target recognition, thereby restricting targetable disease mutations
WT-SpCas9 requires an NGG PAM for target recognition, which restricts targetable disease mutations.
the widely used Streptococcus pyogenes Cas9 (WT-SpCas9) requires an NGG protospacer adjacent motif (PAM) for target recognition, thereby restricting targetable disease mutations
SpCas9-NG recognizes NGN PAMs.
an engineered SpCas9 nuclease variant (SpCas9-NG) recognizing NGN PAMs
SpCas9-NG recognizes NGN PAMs.
an engineered SpCas9 nuclease variant (SpCas9-NG) recognizing NGN PAMs
SpCas9-NG recognizes NGN PAMs.
an engineered SpCas9 nuclease variant (SpCas9-NG) recognizing NGN PAMs
SpCas9-NG recognizes NGN PAMs.
an engineered SpCas9 nuclease variant (SpCas9-NG) recognizing NGN PAMs
SpCas9-NG recognizes NGN PAMs.
an engineered SpCas9 nuclease variant (SpCas9-NG) recognizing NGN PAMs
SpCas9-NG recognizes NGN PAMs.
an engineered SpCas9 nuclease variant (SpCas9-NG) recognizing NGN PAMs
SpCas9-NG recognizes NGN PAMs.
an engineered SpCas9 nuclease variant (SpCas9-NG) recognizing NGN PAMs
SpCas9-NG recognizes NGN PAMs.
an engineered SpCas9 nuclease variant (SpCas9-NG) recognizing NGN PAMs
SpCas9-NG recognizes NGN PAMs.
an engineered SpCas9 nuclease variant (SpCas9-NG) recognizing NGN PAMs
SpCas9-NG recognizes NGN PAMs.
an engineered SpCas9 nuclease variant (SpCas9-NG) recognizing NGN PAMs
SpCas9-NG recognizes NGN PAMs.
an engineered SpCas9 nuclease variant (SpCas9-NG) recognizing NGN PAMs
SpCas9-NG recognizes NGN PAMs.
an engineered SpCas9 nuclease variant (SpCas9-NG) recognizing NGN PAMs
SpCas9-NG recognizes NGN PAMs.
an engineered SpCas9 nuclease variant (SpCas9-NG) recognizing NGN PAMs
SpCas9-NG recognizes NGN PAMs.
an engineered SpCas9 nuclease variant (SpCas9-NG) recognizing NGN PAMs
SpCas9-NG recognizes NGN PAMs.
an engineered SpCas9 nuclease variant (SpCas9-NG) recognizing NGN PAMs
SpCas9-NG recognizes NGN PAMs.
an engineered SpCas9 nuclease variant (SpCas9-NG) recognizing NGN PAMs
SpCas9-NG recognizes NGN PAMs.
an engineered SpCas9 nuclease variant (SpCas9-NG) recognizing NGN PAMs
Approval Evidence
1 source4 linked approval claimsfirst-pass slug spcas9-ng
we recently reported an engineered SpCas9 nuclease variant (SpCas9-NG) recognizing NGN PAMs
Source:
application potentialsupports
SpCas9-NG can be a powerful tool for repairing abnormally expanded CAG repeats and other disease mutations that are difficult to access with WT-SpCas9.
Our study shows that SpCas9-NG can be a powerful tool for repairing abnormally expanded CAG repeats as well as other disease mutations that are difficult to access with WT-SpCas9.
Source:
editing outcomesupports
Targeting the boundary of CAG repeats with SpCas9-NG precisely contracted abnormally expanded CAG repeat tracts in HD-mouse-derived embryonic stem cells.
By targeting the boundary of CAG repeats with SpCas9-NG, we precisely contracted the repeat tracts in HD-mouse-derived embryonic stem (ES) cells.
Source:
phenotypic recoverysupports
Phenotypic abnormalities were recovered in differentiated neurons and in animals produced from repaired ES cells.
we confirmed the recovery of phenotypic abnormalities in differentiated neurons and animals produced from repaired ES cells
Source:
targeting scopesupports
SpCas9-NG recognizes NGN PAMs.
an engineered SpCas9 nuclease variant (SpCas9-NG) recognizing NGN PAMs
Source:
Comparisons
Source-backed strengths
The supplied evidence shows that SpCas9-NG recognizes NGN PAMs and enabled precise contraction of abnormally expanded CAG repeat tracts in HD-mouse-derived embryonic stem cells. The study further reported recovery of phenotypic abnormalities in differentiated neurons and in animals produced from repaired ES cells, supporting functional benefit in that model.
Compared with engineered MT-cleaving enzymes
SpCas9-NG and engineered MT-cleaving enzymes address a similar problem space.
Shared frame: same top-level item type
Strengths here: looks easier to implement in practice.
SpCas9-NG and protein engineering approaches for opto-protein development address a similar problem space.
Shared frame: same top-level item type
Strengths here: looks easier to implement in practice.
SpCas9-NG and synthetic biology approaches for opto-protein development address a similar problem space.
Shared frame: same top-level item type
Strengths here: looks easier to implement in practice.
Ranked Citations
- 1.