Molecular therapy. Methods & clinical development

Consensus-driven target product profiles for curative sickle cell disease gene therapies.

Molecular therapy. Methods & clinical development

Bukini D, Makani J, McCune J, Lee D, Bansbach C, De Vita S, Kemps D, Amin E, Spector J, Tisdale J

Prenatal AAV9-GFP administration in fetal lambs results in transduction of female germ cells and maternal exposure to virus.

Molecular therapy. Methods & clinical development

Borges B, Varthaliti A, Schwab M, Clarke MT, Pivetti C, Gupta N, Cadwell CR, Guibinga G, Phillips S, Del Rio T, Ozsolak F, Imai-Leonard D, Kong L, Laird DJ, Herzeg A, Sumner CJ, MacKenzie TC

Expansion of Human Tregs from Cryopreserved Umbilical Cord Blood for GMP-Compliant Autologous Adoptive Cell Transfer Therapy.

Molecular therapy. Methods & clinical development

Seay HR, Putnam AL, Cserny J, Posgai AL, Rosenau EH, Wingard JR, Girard KF, Kraus M, Lares AP, Brown HL, Brown KS, Balavage KT, Peters LD, Bushdorf AN, Atkinson MA, Bluestone JA, Haller MJ, Brusko TM

Expansion of Human Tregs from Cryopreserved Umbilical Cord Blood for GMP-Compliant Autologous Adoptive Cell Transfer Therapy.

Molecular therapy. Methods & clinical development

Seay HR, Putnam AL, Cserny J, Posgai AL, Rosenau EH, Wingard JR, Girard KF, Kraus M, Lares AP, Brown HL, Brown KS, Balavage KT, Peters LD, Bushdorf AN, Atkinson MA, Bluestone JA, Haller MJ, Brusko TM

Merging DBS with viral vector or stem cell implantation: "hybrid" stereotactic surgery as an evolution in the surgical treatment of Parkinson's disease.

Molecular therapy. Methods & clinical development

Rowland NC, Kalia SK, Kalia LV, Larson PS, Lim DA, Bankiewicz KS

Merging DBS with viral vector or stem cell implantation: "hybrid" stereotactic surgery as an evolution in the surgical treatment of Parkinson's disease.

Molecular therapy. Methods & clinical development

Rowland NC, Kalia SK, Kalia LV, Larson PS, Lim DA, Bankiewicz KS

Maintaining therapeutic activity in the operating room: compatibility of a gamma-retroviral replicating vector with clinical materials and biofluids.

Molecular therapy. Methods & clinical development

Burnett R, Ibañez CE, Pettersson PL, Chen CI, Parab S, Huang T, Robbins J, Bankiewicz K, Aghi M, Logg C, Kasahara N, Pertschuk D, Gruber HE, Jolly DJ

Maintaining therapeutic activity in the operating room: compatibility of a gamma-retroviral replicating vector with clinical materials and biofluids.

Molecular therapy. Methods & clinical development

Burnett R, Ibañez CE, Pettersson PL, Chen CI, Parab S, Huang T, Robbins J, Bankiewicz K, Aghi M, Logg C, Kasahara N, Pertschuk D, Gruber HE, Jolly DJ