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Table 2 Studies reporting therapeutic potential of PTBP1 knockdown

From: Controversies and insights into PTBP1-related astrocyte-neuron transdifferentiation: neuronal regeneration strategies for Parkinson’s and Alzheimer’s disease

Research group & reference

Intended target cell population

Intended CNS region

Animal model(s)

Targeting vector(s)

Lineage tracing

Detection method

Target sequence(s)

Improvements in animal model?

X.D-Fu (Qian et al. (2020))

[25]

Astrocytes

Striatum

PD (6-OHDA) mice

AAV-CMV-LSL-RFP-shPtbp1

mGfap-Cre

Cre-dependent viral reporter

5ʹ-GGGTGAAGATCCTGTTCAATA-3ʹ

(exon 10 of mouse Ptbp1)

Yes

Substantia nigra pars compacta

PD (6-OHDA) mice

Anti-Ptbp1 ASO

hGFAP-CreERT2;R26R-tdTomato

Lineage reporter

5ʹ-GGGTGAAGATCCTGTTCAATA-3ʹ(exon 10 of mouse Ptbp1)

Yes

H.Yang (Zhou et al. (2020))

[27]

Astrocytes

Striatum

PD (6-OHDA)

AAV-GFAP-CasRX-Ptbp1

Not done

Viral reporter

All guide RNAs can be found in Methods section

Yes

Müller glia

Retina

NMDA injured mice

AAV-GFAP-CasRX-Ptbp1

Not done

Viral Cre-induced reporter

All guide RNAs can be found in Methods section

D.W. Cleveland (Maimon et al. (2021))

[24]

Radial glial cells

Hippocampus

Aged WT mice

Anti-Ptbp1 ASO1

hGFAP-CreERT2;R26R-tdTomato

Lineage reporter

5ʹ-TGCGACATTTCTCTGCACTC-3ʹ(3ʹ UTR of mouse Ptbp1)

Yes

Anti-Ptbp1 ASO2

hGFAP-CreERT2;R26R-tdTomato

Lineage reporter

5ʹ-GTGGAAATATTGCTAGGCAC-3ʹ

(5ʹ UTR of mouse Ptbp1)

G.Chen (Yang et al. (2023))

[26]

Spinal astrocytes

Spinal cord

Mouse model of compression-induced SCI

AAV-shPTB: AAV-GFAP-EGFP-shRNA(Ptbp1)-MIR155

Not done

Viral reporter

Same seqeuence as Qian et al. (2020)

Yes

Anti-Ptbp1 ASO (same sequence as Qian et al. (2020))

Not done

Neuronal density

Same seqeuence as Qian et al. (2020)

Yes

E.Huan (Yuan et al. (2024))

[78]

Astrocytes

Cerebral cortex

Mouse model of ischemic stroke

AAV-PHP.eB-GFAP-miR30-shptbp1

Not done

Viral reporter

Same seqeuence as Qian et al. (2020)

Yes

D.W.Cleveland (Maimon et al. (2024) preprint)

[77]

Radial glial cells

Subventricular zone and hippocampus

Young and aged WT mice

Anti-Ptbp1 ASO2 (same one from Maimon et al. (2021))

EdU labelling

MERFISH spatial transcriptomics

5ʹ-GTGGAAATATTGCTAGGCAC-3ʹ

(5ʹ UTR of mouse Ptbp1)

Yes

  1. Table adapted from Wang & Zhang (2023) [62]. 6-OHDA, 6-hydroxydopamine; AAV, adeno-associated virus; ASO, antisense oligonucleotide; MERFISH, Multiplexed error-robust fluorescence in situ hybridization; NMDA, N-methyl-d-aspartate; WT, wild type