Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease.

Kim J, Hu C, Moufawad El Achkar C, Black LE, Douville J, Larson A, Pendergast MK, Goldkind SF, Lee EA, Kuniholm A, Soucy A, Vaze J, Belur NR, Fredriksen K, Stojkovska I, Tsytsykova A, Armant M, DiDonato RL, Choi J, Cornelissen L, Pereira LM, Augustine EF, Genetti CA, Dies K, Barton B, Williams L, Goodlett BD, Riley BL, Pasternak A, Berry ER, Pflock KA, Chu S, Reed C, Tyndall K, Agrawal PB, Beggs AH, Grant PE, Urion DK, Snyder RO, Waisbren SE, Poduri A, Park PJ, Patterson A, Biffi A, Mazzulli JR, Bodamer O, Berde CB, Yu TW

Open source

DOI: 10.1056/nejmoa1813279
Published: 2019 Oct 24
Container: The New England journal of medicine
Publisher: Not recorded
Open access: yes

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Cite this work

BibTeX

@article{allodium:10.1056/nejmoa1813279,
  title = {Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease.},
  author = {Kim J and Hu C and Moufawad El Achkar C and Black LE and Douville J and Larson A and Pendergast MK and Goldkind SF and Lee EA and Kuniholm A and Soucy A and Vaze J and Belur NR and Fredriksen K and Stojkovska I and Tsytsykova A and Armant M and DiDonato RL and Choi J and Cornelissen L and Pereira LM and Augustine EF and Genetti CA and Dies K and Barton B and Williams L and Goodlett BD and Riley BL and Pasternak A and Berry ER and Pflock KA and Chu S and Reed C and Tyndall K and Agrawal PB and Beggs AH and Grant PE and Urion DK and Snyder RO and Waisbren SE and Poduri A and Park PJ and Patterson A and Biffi A and Mazzulli JR and Bodamer O and Berde CB and Yu TW},
  year = {2019},
  journal = {The New England journal of medicine},
  doi = {10.1056/nejmoa1813279},
  url = {https://doi.org/10.1056/nejmoa1813279}
}

RIS

TY  - JOUR
TI  - Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease.
AU  - Kim J
AU  - Hu C
AU  - Moufawad El Achkar C
AU  - Black LE
AU  - Douville J
AU  - Larson A
AU  - Pendergast MK
AU  - Goldkind SF
AU  - Lee EA
AU  - Kuniholm A
AU  - Soucy A
AU  - Vaze J
AU  - Belur NR
AU  - Fredriksen K
AU  - Stojkovska I
AU  - Tsytsykova A
AU  - Armant M
AU  - DiDonato RL
AU  - Choi J
AU  - Cornelissen L
AU  - Pereira LM
AU  - Augustine EF
AU  - Genetti CA
AU  - Dies K
AU  - Barton B
AU  - Williams L
AU  - Goodlett BD
AU  - Riley BL
AU  - Pasternak A
AU  - Berry ER
AU  - Pflock KA
AU  - Chu S
AU  - Reed C
AU  - Tyndall K
AU  - Agrawal PB
AU  - Beggs AH
AU  - Grant PE
AU  - Urion DK
AU  - Snyder RO
AU  - Waisbren SE
AU  - Poduri A
AU  - Park PJ
AU  - Patterson A
AU  - Biffi A
AU  - Mazzulli JR
AU  - Bodamer O
AU  - Berde CB
AU  - Yu TW
PY  - 2019
JO  - The New England journal of medicine
DO  - 10.1056/nejmoa1813279
UR  - https://doi.org/10.1056/nejmoa1813279
ER  -

APA

J, K., C, H., C, M. E. A., LE, B., J, D., A, L., MK, P., SF, G., EA, L., A, K., A, S., J, V., NR, B., K, F., I, S., A, T., M, A., RL, D., J, C., L, C., LM, P., EF, A., CA, G., K, D., B, B., L, W., BD, G., BL, R., A, P., ER, B., KA, P., S, C., C, R., K, T., PB, A., AH, B., PE, G., DK, U., RO, S., SE, W., A, P., PJ, P., A, P., A, B., JR, M., O, B., CB, B., & TW, Y. (2019). Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease.. The New England journal of medicine. https://doi.org/10.1056/nejmoa1813279

Source records

pubmed · retrieved 2026-09-27T06:21:56.035Z