RNA interference approaches for treatment of HIV-1 infection

Bobbin, Maggie L; Burnett, John C; Rossi, John J

doi:10.1186/s13073-015-0174-y

Table 1 Current therapies, clinical trials, and strategies to combat HIV

From: RNA interference approaches for treatment of HIV-1 infection

Therapeutic approach	Company/institute	Component(s)	Target(s)	Delivery	Progress	NCT identifier	Reference(s)
Combinatorial anti-retroviral therapy (cART)	Gilead, Merck, GSK, Pfizer, Tibotec, Abbott, Bristol-Meyers Squibb, Hoffman-La Roche	1. CCR5 antagonist	1. Receptor binding	In vivo drug administration	Current method to treat HIV		[159]
		2. Fusion inhibitors	2. Membrane fusion
		3. Nucleoside analogs or RT inhibitors	3. Reverse transcription
		4. Strand transfer inhibitor	4. Integration
		5. Protease inhibitors	5. Protein processing
Peptide or protein vaccines	VaxGen, Merck, NIH/NIAID	Gp120	Viral envelope	In vivo injection	Phase I/II/III	NCT00001031	[160–162]
		Gp41/160				NCT00002402
		Gag/pol/nef	Viral proteins			NCT00223080
						NCT01435135
						NCT00002441
Inhibitory peptide	University Medical Center Hamburg-Eppendorf	C46 anti-viral peptide	Viral fusion to cell membrane	Ex vivo retroviral modified CD4+ cells	Phase I		[163]
Drug shRNA peptide	Calimmune/Caltech/UCLA	1. Bisulfide	1. Transplant conditioning	Ex vivo lentiviral modified CD34+ and CD4+ cells	Phase I/II	NCT01734850	[139]
		2. CCR5 shRNA	2. Host co-receptor
		3. C46 peptide	3. Viral envelope
shRNA ribozyme RNA decoy	Benitec/City of Hope	1. tat/rev shRNA	1. Viral mRNA	Ex vivo lentiviral modified CD34+ cells	Phase I	NCT00569985	[38, 39]
		2. CCR5 ribozyme	2. Host co-receptor
		3. TAR decoy	3. Viral Tat protein
Zinc finger nuclease	Sangamo Biosciences	1. CCR5 zinc finger nuclease	CCR5 DNA	Ex vivo AAV modified autologous CD4+ T cells	Phase I-II	NCT00842634	[20]
						NCT01044654
						NCT01543152
						NCT02225665
Ribozyme	Janssen-Cilag Pty Ltd, UCLA	tat-vpr anti-HIV ribozyme	Tat-vpr mRNA	Ex vivo retroviral modified autologous CD34+ HPC	Phase I/II	NCT00074997	[40, 41]
						NCT01177059
Ribozyme	UCSD	CCR5 ribozyme	CCR5 mRNA	Ex vivo retroviral modified autologous CD4+ T cells	Phase I	FDA BB-IND 6405	[42]
Ribozyme	Johnson & Johnson, St Vincent’s Hospital	Tat anti-HIV ribozyme	Translation initiation mRNA of Tat	Ex vivo retroviral modified syngeneic CD4+ T cells	Phase I	NCT00074997	[43]
Ribozyme	Ribozyme, City of Hope	Tat anti-HIV ribozyme	Tat-rev mRNA	Ex vivo retroviral modified CD34+ HPC	Phase II	NCT00002221	[164]
Antisense	VIRxSYS Corporation	937 base antisense gene	Env mRNA	Ex vivo lentiviral (LTR HIV) modified CD4+ cells	Phase I/II	NCT00131560	[165]
Antisense	Enzo Biochem	U1/HIV anti-sense RNA (61–68 bases)	TAR, tat/rev	Ex vivo retroviral modified CD34+ HPC	Phase I/II	NCT00003942	[166]
						NCT00001535
RNA decoy	Children’s Hospital Los Angeles	Rev response element decoy	Rev protein	Ex vivo retroviral modified CD34+ HPC	Phase 0- pilot		[167]

Abbreviations: AAV, adeno-associated virus; HPC, human progenitor cells; LTR, long terminal repeat; NCT, National Clinical Trial; NIH/NIAID, National Institutes of Health/National Institute of Allergy and Infectious Diseases; RT, reverse transcriptase; shRNA, short hairpin RNA

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ISSN: 1756-994X

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