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Characterization of Functionally Associated miRNAs in Glioblastoma and their Engineering into Artificial Clusters for Gene Therapy
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Effects of APT20TTMG, a modulator of the U1 snRNP complex, in glioblastoma models

Caio Bruno Quinta de Souza Leal1, Camila Guimarães Moreira Zimmer1, Vanessa de Vasconcelos Castilho Sinatti1

  • 1Aptah Bio Inc., 930 Brittan Avenue, San Carlos, CA, 94070, USA.

Medical Oncology (Northwood, London, England)
|October 2, 2025
PubMed

Insights

A novel compound, APT20TTMG, effectively targets U1 small nuclear ribonucleoprotein (snRNP) complex dysfunction in glioblastoma. This therapeutic strategy shows promise in reducing tumor growth and improving outcomes for cancer patients.

Area of Science:

  • Molecular Biology
  • Oncology
  • Biochemistry

Background:

  • The U1 small nuclear ribonucleoprotein (snRNP) complex plays a critical role in pre-mRNA splicing and preventing premature polyadenylation.
  • Dysfunction of the U1 snRNP complex is linked to oncogenic splicing and tumor progression in cancers like glioblastoma.

Purpose of the Study:

  • To investigate the therapeutic potential of APT20TTMG, a synthetic cDNA designed to modulate U1 snRNP misassembly, for glioblastoma treatment.
  • To assess APT20TTMG's internalization, in vitro effects on glioblastoma cells, and in vivo efficacy in a mouse model.

Main Methods:

  • Assessed APT20TTMG internalization in U-87 MG cells.
  • Evaluated in vitro effects on cell viability, proliferation, and apoptosis.
  • Administered APT20TTMG intravenously in athymic mice to assess tumor-related parameters and combined therapy with temozolomide.

Main Results:

  • APT20TTMG showed over 50% internalization and demonstrated cytotoxic, cytostatic, and pro-apoptotic effects in vitro.
  • In vivo, APT20TTMG treatment reduced tumor volume, slowed growth, and improved body weight trends over 22 days.
  • Treatment decreased oncogenic pathways, enhanced histopathological outcomes, and showed improved antitumor efficacy when combined with temozolomide.

Conclusions:

  • APT20TTMG effectively corrects U1 snRNP complex dysfunction in glioblastoma models.
  • This compound holds significant potential as a therapeutic strategy for modulating splicing in glioblastoma.
Keywords:
Antitumor therapyGlioblastomaSubcutaneous xenograft modelU-87 MGU1 snRNP complex

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