What question did this study set out to answer?

To evaluate whether targeting CD117 with an antibody-drug conjugate can selectively deplete host hematopoietic stem cells to facilitate donor engraftment without the genotoxicity of traditional conditioning regimens.

February 6, 2019Nature CommunicationsOpen Access

Selective hematopoietic stem cell ablation using CD117-antibody-drug-conjugates enables safe and effective transplantation with immunity preservation

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Authors

ACAgnieszka Czechowicz RPRahul Palchaudhuri ASAmelia Scheck

Discussion

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Overview

Preclinical study shows CD117-antibody-drug conjugates safely clear host stem cells while preserving immune defenses, indicating a safer conditioning path for transplantation and gene therapies.

Key Points

To evaluate whether targeting CD117 with an antibody-drug conjugate can selectively deplete host hematopoietic stem cells to facilitate donor engraftment without the genotoxicity of traditional conditioning regimens.
Administered a single dose of a CD117-antibody-drug conjugate carrying the ribotoxin saporin (CD117-ADC) as pre-transplant conditioning.
Assessed host hematopoietic stem cell depletion, donor engraftment efficiency, adverse side effects, and immune competence against viral and fungal challenges.
A single dose of CD117-ADC depleted >99% of host hematopoietic stem cells, allowing rapid and efficient donor cell engraftment.
Treatment demonstrated high target selectivity without clinically significant toxicity, maintaining baseline blood counts and protective immune responses against both viral and fungal pathogens.

Cite This Study

Czechowicz et al. (2019) studied this question.

synapsesocial.com/papers/6a1b74fa0ea968f653abde89 https://doi.org/10.1038/s41467-018-08201-x

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February 6, 2019Nature CommunicationsOpen Access

Selective hematopoietic stem cell ablation using CD117-antibody-drug-conjugates enables safe and effective transplantation with immunity preservation

View Full Paper

Ask AI

Bookmark

Authors

ACAgnieszka Czechowicz RPRahul Palchaudhuri ASAmelia Scheck

Discussion

Member takes

Overview

Preclinical study shows CD117-antibody-drug conjugates safely clear host stem cells while preserving immune defenses, indicating a safer conditioning path for transplantation and gene therapies.

Key Points

To evaluate whether targeting CD117 with an antibody-drug conjugate can selectively deplete host hematopoietic stem cells to facilitate donor engraftment without the genotoxicity of traditional conditioning regimens.
Administered a single dose of a CD117-antibody-drug conjugate carrying the ribotoxin saporin (CD117-ADC) as pre-transplant conditioning.
Assessed host hematopoietic stem cell depletion, donor engraftment efficiency, adverse side effects, and immune competence against viral and fungal challenges.
A single dose of CD117-ADC depleted >99% of host hematopoietic stem cells, allowing rapid and efficient donor cell engraftment.
Treatment demonstrated high target selectivity without clinically significant toxicity, maintaining baseline blood counts and protective immune responses against both viral and fungal pathogens.

Cite This Study

Czechowicz et al. (2019) studied this question.

synapsesocial.com/papers/6a1b74fa0ea968f653abde89 https://doi.org/10.1038/s41467-018-08201-x

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