Leillani L Ha

8PUBLICATIONS
48CO-AUTHORS
Neurology and neuromuscular diseasesGene and molecular therapyProcess control and simulationMedical biochemistry - proteins and peptides (incl. medical proteomics)
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Publications (8)

|Jul 15, 2026
Managing nusinersen therapy during pregnancy in spinal muscular atrophy type 3.

|Apr 03, 2026
Cardiac defects in spinal muscular atrophy and the role of SMN in cardiomyocyte homeostasis.

Reid Garner, Leillani L Ha, Flavia C Nery

|Jan 29, 2026
Circulating Tau Profiles in Pediatric and Adult Patients with Spinal Muscular Atrophy.

Leillani L Ha, Sunayana Mitra, Doreen T Ho

|Sep 11, 2025
Treatment of a severe vascular disease using a bespoke CRISPR-Cas9 base editor in mice.

Christiano R R Alves, Sabyasachi Das, Vijai Krishnan

|Apr 22, 2025
Custom CRISPR-Cas9 PAM variants via scalable engineering and machine learning.

Rachel A Silverstein, Nahye Kim, Ann-Sophie Kroell

|Apr 01, 2025
Early life safety profiling of gene therapy for spinal muscular atrophy.

Rebecca G Spellman, Leillani L Ha, Salomé Da Silva Duarte Lepez

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Frequent Collaborators

4 joint publications

Christiano R R Alves

4 joint publications

Benjamin P Kleinstiver

3 joint publications

Shengdar Q Tsai

3 joint publications

Luke H Chao