# Therapeutic Gene Editing of the Beta-Hemoglobin Locus by Homology Directed Repair

> **NIH NIH K08** · SEATTLE CHILDREN'S HOSPITAL · 2020 · $128,694

## Abstract

PROJECT SUMMARY / ABSTRACT
This following project is an NIH Mentored Clinical Scientist Research Career Development Award (K08)
application for Dr. Christopher Lux, an Acting Instructor in the Department of Pediatrics at the University of
Washington (UW). Dr. Lux has completed his clinical training in Pediatric Hematology Oncology and Bone
Marrow Transplant and is committed to a career as a physician-scientist focused on the treatment of
hemoglobinopathy patients. Dr. Lux's specific research interest is the generation and testing of gene editing
tools to modify the hemoglobin locus within hematopoietic stem cells for autologous transplant. His long-term
career goal is to establish himself as an independently-funded principle investigator developing gene therapy
solutions for patients and aiding in their ethical and safe introduction to clinical use.
In order to achieve this goal, Dr. Lux is requesting NIH K08 support for additional training and mentorship in
the following specific areas: (1) additional training in gene editing of human and non-human primate
hematopoietic stem cells and repair template design and delivery; (2) additional training in bioethics as it
relates to emerging gene editing technologies; (3) additional training in clinical trials design and
implementation; (4) attendance of scientific conferences, career development seminars, and additional
classroom-based training relevant to this project; and (5) mentorship regarding successful transition to
scientific independence.
Dr. Lux proposes research efforts to achieve further his initial progress in developing a therapeutic gene editing
approach for the treatment of hemoglobinopathy patients including: Aim 1) developing procedures to express
therapeutic levels of hemoglobin by site specific modification of the γ-hemoglobin promoter using homology-
directed-repair (HDR) donor templates; Aim 2) assessing the ability of HDR edited human cells to engraft in
immunodeficient mice and to allow for chemo-selection; and Aim 3: utilizing the non-human primate system to
assess the potential for HDR edited CD34+ cells to approximate the clinical performance of these tools without
a xenograft barrier. Aims 1&2 will be carried out primarily at the Seattle Children's Research Institute under
the mentorship of Dr. David Rawlings. Aim 3 will be carried out primarily at the Fred Hutchinson Cancer
Research Center under the mentorship of Dr. Hans-Peter Kiem. Both mentors have significant track records
for training physician scientists and developing new therapies. The proposed studies offer a potential path to
site specific gene editing and chemo-selection as a means for a treating the most common single gene
disorders in man. These studies will be expected to yield publications and the preliminary data required for
independent R01 funding.

## Key facts

- **NIH application ID:** 9951799
- **Project number:** 1K08HL151814-01
- **Recipient organization:** SEATTLE CHILDREN'S HOSPITAL
- **Principal Investigator:** Christopher Lux
- **Activity code:** K08 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $128,694
- **Award type:** 1
- **Project period:** 2020-04-15 → 2020-05-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9951799

## Citation

> US National Institutes of Health, RePORTER application 9951799, Therapeutic Gene Editing of the Beta-Hemoglobin Locus by Homology Directed Repair (1K08HL151814-01). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/9951799. Licensed CC0.

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